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Tytuł :
Physiologically based pharmacokinetic (PBPK) modeling of RNAi therapeutics: Opportunities and challenges.
Autorzy :
Fairman K; Division of Biochemical Toxicology, National Center for Toxicological Research, United States Food and Drug Administration, Jefferson, AR 72079, USA.
Li M; Division of Biochemical Toxicology, National Center for Toxicological Research, United States Food and Drug Administration, Jefferson, AR 72079, USA.
Ning B; Division of Bioinformatics and Biostatistics, National Center for Toxicological Research, United States Food and Drug Administration, Jefferson, AR 72079, USA.
Lumen A; Division of Biochemical Toxicology, National Center for Toxicological Research, United States Food and Drug Administration, Jefferson, AR 72079, USA. Electronic address: .
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Źródło :
Biochemical pharmacology [Biochem Pharmacol] 2021 Jul; Vol. 189, pp. 114468. Date of Electronic Publication: 2021 Feb 10.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Review
MeSH Terms :
Models, Biological*
Drug Development/*methods
RNA Interference/*drug effects
RNAi Therapeutics/*methods
Animals ; Antibodies, Monoclonal/genetics ; Antibodies, Monoclonal/metabolism ; Drug Development/trends ; Humans ; Oligonucleotides/antagonists & inhibitors ; Oligonucleotides/genetics ; Oligonucleotides/metabolism ; RNA Interference/physiology ; RNAi Therapeutics/trends
Czasopismo naukowe
Tytuł :
Innovations in Biomaterial Design toward Successful RNA Interference Therapy for Cancer Treatment.
Autorzy :
Ward DM; McKetta Department of Chemical Engineering, 200 E. Dean Keeton St. Stop C0400, Austin, TX, 78712, USA.; Institute for Biomaterials, Drug Delivery, and Regenerative Medicine, The University of Texas at Austin, 107 W Dean Keeton Street Stop C0800, Austin, TX, 78712, USA.
Shodeinde AB; McKetta Department of Chemical Engineering, 200 E. Dean Keeton St. Stop C0400, Austin, TX, 78712, USA.; Institute for Biomaterials, Drug Delivery, and Regenerative Medicine, The University of Texas at Austin, 107 W Dean Keeton Street Stop C0800, Austin, TX, 78712, USA.
Peppas NA; McKetta Department of Chemical Engineering, 200 E. Dean Keeton St. Stop C0400, Austin, TX, 78712, USA.; Institute for Biomaterials, Drug Delivery, and Regenerative Medicine, The University of Texas at Austin, 107 W Dean Keeton Street Stop C0800, Austin, TX, 78712, USA.; Department of Biomedical Engineering, The University of Texas at Austin, 107 W Dean Keeton Street Stop C0800, Austin, TX, 78712, USA.; Division of Molecular Pharmaceutics and Drug Delivery, College of Pharmacy, The University of Texas at Austin, 2409 University Ave. Stop A1900, Austin, TX, 78712, USA.; Department of Pediatrics and Department of Surgery and Perioperative Care, Dell Medical School, 1601 Trinity St., Bldg. B, Stop Z0800, Austin, TX, 78712, USA.
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Źródło :
Advanced healthcare materials [Adv Healthc Mater] 2021 Jul; Vol. 10 (13), pp. e2100350. Date of Electronic Publication: 2021 May 11.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review
MeSH Terms :
Neoplasms*/genetics
Neoplasms*/therapy
RNAi Therapeutics*
Biocompatible Materials ; Drug Carriers ; Nanotechnology ; RNA Interference ; RNA, Small Interfering/genetics
Czasopismo naukowe
Tytuł :
How Should Clinicians Execute Critical Force Interventions With Compassion, Not Just Harm Minimization, as a Clinical and Ethical Goal?
Autorzy :
Trestman RL; Professor and chair of psychiatry at the Virginia Tech Carilion School of Medicine and chair of psychiatry and behavioral medicine at Carilion Clinic in Roanoke, Virginia.
Nagaraja K; Resident physician in the Department of Psychiatry and Behavioral Medicine at Carilion Clinic in Roanoke, Virginia.
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Źródło :
AMA journal of ethics [AMA J Ethics] 2021 Apr 01; Vol. 23 (4), pp. E292-297. Date of Electronic Publication: 2021 Apr 01.
Typ publikacji :
Case Reports; Journal Article
MeSH Terms :
Empathy*
Harm Reduction*
Mental Disorders*/therapy
Therapeutics*/ethics
Therapeutics*/psychology
Adult ; Health Personnel/ethics ; Humans ; Mental Health Services/ethics ; Mental Health Services/trends ; Motivation ; Self-Injurious Behavior/therapy
Czasopismo naukowe
Tytuł :
Theragnostics: A Neologism That Generates Schism-The Hellenic Thesis.
Autorzy :
Frangos S; Bank of Cyprus Oncology CenterCyprus, GreeceE-mail: .
Giannoula E
Buscombe J
Iakovou I
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Źródło :
Journal of nuclear medicine : official publication, Society of Nuclear Medicine [J Nucl Med] 2021 Jun 01; Vol. 62 (6), pp. 880. Date of Electronic Publication: 2021 Mar 26.
Typ publikacji :
Letter
MeSH Terms :
Diagnosis*
Terminology as Topic*
Therapeutics*
Opinia redakcyjna
Tytuł :
In Vitro Inhibitory Analysis of Rationally Designed siRNAs against MERS-CoV Replication in Huh7 Cells.
Autorzy :
El-Kafrawy SA; Special Infectious Agents Unit, King Fahd Medical Research Center, King Abdulaziz University, Jeddah 21589, Saudi Arabia.; Department of Medical Laboratory Technology, Faculty of Applied Medical Sciences, King Abdulaziz University, Jeddah 21589, Saudi Arabia.
Sohrab SS; Special Infectious Agents Unit, King Fahd Medical Research Center, King Abdulaziz University, Jeddah 21589, Saudi Arabia.; Department of Medical Laboratory Technology, Faculty of Applied Medical Sciences, King Abdulaziz University, Jeddah 21589, Saudi Arabia.
Mirza Z; Department of Medical Laboratory Technology, Faculty of Applied Medical Sciences, King Abdulaziz University, Jeddah 21589, Saudi Arabia.; King Fahd Medical Research Center, King Abdulaziz University, Jeddah 21589, Saudi Arabia.
Hassan AM; Special Infectious Agents Unit, King Fahd Medical Research Center, King Abdulaziz University, Jeddah 21589, Saudi Arabia.
Alsaqaf F; Special Infectious Agents Unit, King Fahd Medical Research Center, King Abdulaziz University, Jeddah 21589, Saudi Arabia.
Azhar EI; Special Infectious Agents Unit, King Fahd Medical Research Center, King Abdulaziz University, Jeddah 21589, Saudi Arabia.; Department of Medical Laboratory Technology, Faculty of Applied Medical Sciences, King Abdulaziz University, Jeddah 21589, Saudi Arabia.
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Źródło :
Molecules (Basel, Switzerland) [Molecules] 2021 Apr 29; Vol. 26 (9). Date of Electronic Publication: 2021 Apr 29.
Typ publikacji :
Journal Article
MeSH Terms :
RNAi Therapeutics*
Virus Replication*/drug effects
Coronavirus Infections/*therapy
Middle East Respiratory Syndrome Coronavirus/*physiology
RNA, Small Interfering/*pharmacology
Cell Line ; Coronavirus Infections/virology ; Humans ; Middle East Respiratory Syndrome Coronavirus/drug effects ; Middle East Respiratory Syndrome Coronavirus/genetics ; RNA, Small Interfering/administration & dosage ; RNA, Small Interfering/genetics ; Viral Proteins/genetics
Czasopismo naukowe
Tytuł :
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1.
Autorzy :
Garrelfs SF; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Frishberg Y; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Hulton SA; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Koren MJ; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
O'Riordan WD; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Cochat P; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Deschênes G; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Shasha-Lavsky H; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Saland JM; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Van't Hoff WG; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Fuster DG; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Magen D; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Moochhala SH; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Schalk G; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Simkova E; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Groothoff JW; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Sas DJ; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Meliambro KA; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Lu J; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Sweetser MT; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Garg PP; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Vaishnaw AK; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Gansner JM; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
McGregor TL; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
Lieske JC; From the Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam (S.F.G., J.W.G.); the Division of Pediatric Nephrology, Shaare Zedek Medical Center, Jerusalem (Y.F.); the Department of Nephrology, Birmingham Women's and Children's Hospital, Birmingham (S.A.H.), and the Department of Paediatric Nephrology, Great Ormond Street Hospital (W.G.H.), and UCL Department of Renal Medicine, Royal Free Hospital (S.H.M.), London - both in the United Kingdom; Jacksonville Center for Clinical Research, Jacksonville, FL (M.J.K.); eStudySite, San Diego, CA (W.D.O.); Center for Rare Renal Diseases and INSERM Pediatric Clinical Investigation Center-Hospices Civils de Lyon and Université de Lyon, Lyon (P.C.), and the Department of Pediatric Nephrology, Hôpital Robert-Debré, Paris (G.D.) - both in France; the Pediatric Nephrology Unit, Galilee Medical Center, Nahariya (H.S.-L.), and the Pediatric Nephrology Institute, Rambam Health Care Campus, Haifa (D.M.) - both in Israel; the Icahn School of Medicine at Mount Sinai, New York (J.M.S., K.A.M.); the Department of Nephrology and Hypertension, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland (D.G.F.); the University of Bonn, Bonn, Germany (G.S.); Al Jalila Children's Hospital, Dubai, United Arab Emirates (E.S.); the Divisions of Pediatric Nephrology and Hypertension (D.J.S.) and Nephrology and Hypertension (J.C.L.), Mayo Clinic, Rochester, MN; and Alnylam Pharmaceuticals, Cambridge, MA (J.L., M.T.S., P.P.G., A.K.V., J.M.G., T.L.M.).
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Corporate Authors :
ILLUMINATE-A Collaborators
Źródło :
The New England journal of medicine [N Engl J Med] 2021 Apr 01; Vol. 384 (13), pp. 1216-1226.
Typ publikacji :
Clinical Trial, Phase III; Journal Article; Multicenter Study; Randomized Controlled Trial; Research Support, Non-U.S. Gov't
MeSH Terms :
RNAi Therapeutics*
Hyperoxaluria, Primary/*drug therapy
Oxalates/*urine
RNA, Small Interfering/*therapeutic use
Adolescent ; Adult ; Child ; Creatinine/urine ; Double-Blind Method ; Female ; Glomerular Filtration Rate ; Humans ; Hyperoxaluria, Primary/blood ; Hyperoxaluria, Primary/complications ; Hyperoxaluria, Primary/urine ; Kidney Calculi/prevention & control ; Male ; Middle Aged ; Oxalates/blood ; Oxalates/metabolism ; RNA, Small Interfering/adverse effects ; Young Adult
SCR Disease Name :
Primary hyperoxaluria type 1
Czasopismo naukowe
Tytuł :
Multifunctional Nanoprobe for the Delivery of Therapeutic siRNA and Real-Time Molecular Imaging of Parkinson's Disease Biomarkers.
Autorzy :
Li R; Department of Biomedical Engineering, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Li Y; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Mu M; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Yang B; Department of Biomedical Engineering, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Chen X; Department of Biomedical Engineering, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Lee WYW; Department of Orthopedic and Traumatology, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Ke Y; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Yung WH; School of Biomedical Sciences, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.
Tang BZ; Hong Kong Branch of Chinese National Engineering Research Center for Tissue Restoration and Reconstruction, Department of Chemistry, The Hong Kong University of Science and Technology, Clear Water Bay, Kowloon, 999077 Hong Kong, P. R. China.
Bian L; Department of Biomedical Engineering, The Chinese University of Hong Kong, Shatin, New Territories, 999077 Hong Kong, P. R. China.; The Chinese University of Hong Kong Shenzhen Research Institute, Shenzhen 518000, China.
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Źródło :
ACS applied materials & interfaces [ACS Appl Mater Interfaces] 2021 Mar 17; Vol. 13 (10), pp. 11609-11620. Date of Electronic Publication: 2021 Mar 08.
Typ publikacji :
Journal Article
MeSH Terms :
RNAi Therapeutics*
Parkinson Disease/*diagnostic imaging
Parkinson Disease/*therapy
Peptides/*chemistry
RNA, Small Interfering/*administration & dosage
Cell Line ; Gene Transfer Techniques ; Humans ; Matrix Metalloproteinase 3/analysis ; Matrix Metalloproteinase 3/genetics ; Molecular Imaging ; Parkinson Disease/genetics ; RNA, Small Interfering/genetics ; RNA, Small Interfering/therapeutic use
Czasopismo naukowe
Tytuł :
Squalenoyl siRNA PMP22 nanoparticles are effective in treating mouse models of Charcot-Marie-Tooth disease type 1 A.
Autorzy :
Boutary S; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Caillaud M; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
El Madani M; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.; National Research Centre, Cairo, Egypt.
Vallat JM; Service de Neurologie - Centre de Référence Neuropathies Périphérique Rares, CHU de Limoges - Hôpital Dupuytren, 2 Avenue Martin Luther King, 87042, LIMOGES CEDEX, France.
Loisel-Duwattez J; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.; Neurology Department, AP-HP, Université Paris-Saclay and French Reference Center for Familial Amyloid Polyneuropathy and other rare peripheral neuropathies (CRMR-NNERF), Bicêtre University Hospital, Le Kremlin-Bicêtre, France.
Rouyer A; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Richard L; Service de Neurologie - Centre de Référence Neuropathies Périphérique Rares, CHU de Limoges - Hôpital Dupuytren, 2 Avenue Martin Luther King, 87042, LIMOGES CEDEX, France.
Gracia C; UMR 8203 CNRS, newly UMR 9018 CNRS, Université Paris-Saclay, 94805, Villejuif, France.
Urbinati G; UMR 8203 CNRS, newly UMR 9018 CNRS, Université Paris-Saclay, 94805, Villejuif, France.
Desmaële D; Institut Galien Paris-Sud, CNRS UMR 8612, Université Paris-Sud, Université Paris-Saclay, 92290, Châtenay-Malabry, France.
Echaniz-Laguna A; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.; Neurology Department, AP-HP, Université Paris-Saclay and French Reference Center for Familial Amyloid Polyneuropathy and other rare peripheral neuropathies (CRMR-NNERF), Bicêtre University Hospital, Le Kremlin-Bicêtre, France.
Adams D; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.; Neurology Department, AP-HP, Université Paris-Saclay and French Reference Center for Familial Amyloid Polyneuropathy and other rare peripheral neuropathies (CRMR-NNERF), Bicêtre University Hospital, Le Kremlin-Bicêtre, France.
Couvreur P; Institut Galien Paris-Sud, CNRS UMR 8612, Université Paris-Sud, Université Paris-Saclay, 92290, Châtenay-Malabry, France.
Schumacher M; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France.
Massaad C; Faculty of Basic and Biomedical Sciences, Paris Descartes University, INSERM UMRS 1124, 75006, Paris, France.
Massaad-Massade L; U1195 Diseases and Hormones of the Nervous System, Inserm and University Paris-Saclay, 94276, Le Kremlin-Bicêtre, France. .
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Źródło :
Communications biology [Commun Biol] 2021 Mar 09; Vol. 4 (1), pp. 317. Date of Electronic Publication: 2021 Mar 09.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't; Video-Audio Media
MeSH Terms :
Gene Transfer Techniques*
Nanoconjugates*
RNA Interference*
RNAi Therapeutics*
Charcot-Marie-Tooth Disease/*therapy
Myelin Proteins/*genetics
Nerve Fibers, Myelinated/*metabolism
RNA, Small Interfering/*genetics
Squalene/*chemistry
Animals ; Cell Line ; Charcot-Marie-Tooth Disease/genetics ; Charcot-Marie-Tooth Disease/metabolism ; Charcot-Marie-Tooth Disease/physiopathology ; Disease Models, Animal ; Early Growth Response Protein 2/genetics ; Early Growth Response Protein 2/metabolism ; Mice, Inbred C57BL ; Mice, Transgenic ; Motor Activity ; Myelin Proteins/metabolism ; Nerve Fibers, Myelinated/pathology ; Nerve Regeneration ; Neurofilament Proteins/genetics ; Neurofilament Proteins/metabolism ; RNA, Small Interfering/metabolism ; Recovery of Function ; SOXE Transcription Factors/genetics ; SOXE Transcription Factors/metabolism ; Time Factors
Czasopismo naukowe
Tytuł :
 
Autorzy :
Lehmkuhl G; Klinik für Psychiatrie, Psychosomatik und Psychotherapie des Kindes- und Jugendalters der Universität zu Köln.
Holtmann M; LWL-Universitätsklinik Hamm der Ruhr-Universität Bochum.
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Transliterated Title :
„Learning from Stories“: Die Bedeutung von Fallgeschichten für Diagnostik und Therapie.
Źródło :
Zeitschrift fur Kinder- und Jugendpsychiatrie und Psychotherapie [Z Kinder Jugendpsychiatr Psychother] 2021 Mar; Vol. 49 (2), pp. 89-91.
Typ publikacji :
Editorial
MeSH Terms :
Diagnosis*
Medical History Taking*
Therapeutics*
Humans
Opinia redakcyjna
Tytuł :
Understanding the antiviral effects of RNAi-based therapy in HBeAg-positive chronic hepatitis B infection.
Autorzy :
Kadelka S; Department of Mathematics, Virginia Tech, Blacksburg, VA, 24060, USA.
Dahari H; Program for Experimental and Theoretical Modeling, Stritch School of Medicine, Loyola University Chicago, Maywood, IL, 60153, USA.
Ciupe SM; Department of Mathematics, Virginia Tech, Blacksburg, VA, 24060, USA. .
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Źródło :
Scientific reports [Sci Rep] 2021 Jan 08; Vol. 11 (1), pp. 200. Date of Electronic Publication: 2021 Jan 08.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, U.S. Gov't, Non-P.H.S.
MeSH Terms :
RNAi Therapeutics*
Hepatitis B e Antigens/*metabolism
Hepatitis B, Chronic/*metabolism
Hepatitis B, Chronic/*therapy
Adult ; Female ; Hepatitis B, Chronic/genetics ; Humans ; Male ; Middle Aged
Czasopismo naukowe
Tytuł :
siRNA Therapeutics for Protein Misfolding Diseases of the Central Nervous System.
Autorzy :
Zabel MD; Prion Research Center, Department of Microbiology, Immunology and Pathology, College of Veterinary Medicine and Biomedical Sciences, Colorado State University, Fort Collins, CO, USA. .
Mollnow L; Prion Research Center, Department of Microbiology, Immunology and Pathology, College of Veterinary Medicine and Biomedical Sciences, Colorado State University, Fort Collins, CO, USA.
Bender H; Prion Research Center, Department of Microbiology, Immunology and Pathology, College of Veterinary Medicine and Biomedical Sciences, Colorado State University, Fort Collins, CO, USA.
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Źródło :
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 377-394.
Typ publikacji :
Journal Article
MeSH Terms :
Gene Transfer Techniques*
RNA Interference*
RNAi Therapeutics*
Central Nervous System Diseases/*therapy
Lipids/*chemistry
Proteins/*chemistry
Proteostasis Deficiencies/*therapy
RNA, Small Interfering/*genetics
Animals ; Blood-Brain Barrier/metabolism ; Central Nervous System Diseases/genetics ; Central Nervous System Diseases/metabolism ; Liposomes ; Mice, Inbred Strains ; Protein Folding ; Proteostasis Deficiencies/genetics ; Proteostasis Deficiencies/metabolism ; RNA, Small Interfering/chemistry ; RNA, Small Interfering/metabolism ; Receptors, Nicotinic/genetics ; Receptors, Nicotinic/metabolism
Czasopismo naukowe
Tytuł :
Production and Characterization of Anti-CCR5 siRNA-Loaded Polycaprolactone Nanoparticles for Topical Pre-exposure Prophylaxis.
Autorzy :
Ribeiro R; i3S-Instituto de Investigação e Inovação em Saúde & INEB-Instituto de Engenharia Biomédica, Universidade do Porto, Porto, Portugal.
Sarmento B; i3S-Instituto de Investigação e Inovação em Saúde & INEB-Instituto de Engenharia Biomédica, Universidade do Porto, Porto, Portugal.; CESPU, Instituto de Investigação e Formação Avançada em Ciências e Tecnologias da Saúde, Gandra, Portugal.
das Neves J; i3S-Instituto de Investigação e Inovação em Saúde & INEB-Instituto de Engenharia Biomédica, Universidade do Porto, Porto, Portugal. .; CESPU, Instituto de Investigação e Formação Avançada em Ciências e Tecnologias da Saúde, Gandra, Portugal. .
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Źródło :
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 403-416.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Gene Transfer Techniques*
Pre-Exposure Prophylaxis*
RNA Interference*
RNAi Therapeutics*
HIV Infections/*prevention & control
HIV-1/*pathogenicity
RNA, Small Interfering/*genetics
Receptors, CCR5/*genetics
Cells, Cultured ; HIV Infections/immunology ; HIV Infections/transmission ; HIV Infections/virology ; HIV-1/immunology ; Humans ; Nanoparticles ; Polyesters/chemistry ; RNA, Small Interfering/metabolism ; Receptors, CCR5/metabolism ; Research Design ; Workflow
Czasopismo naukowe
Tytuł :
Delivery of siRNA to the Eye: Protocol for a Feasibility Study to Assess Novel Delivery System for Topical Delivery of siRNA Therapeutics to the Ocular Surface.
Autorzy :
Baran-Rachwalska P; Biomedical Sciences Research Institute, Ulster University, Coleraine, Northern Ireland, UK.; SiSaf Ltd., Guildford, UK.
Saffie-Siebert S; SiSaf Ltd., Guildford, UK.
Moore CBT; Biomedical Sciences Research Institute, Ulster University, Coleraine, Northern Ireland, UK. .; SiSaf Ltd., Guildford, UK. .; Avellino Lab USA, Inc., Menlo Park, CA, USA. .
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Źródło :
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 443-453.
Typ publikacji :
Journal Article
MeSH Terms :
RNA Interference*
RNAi Therapeutics*
Epithelium, Corneal/*metabolism
Eye Diseases/*therapy
RNA, Small Interfering/*genetics
Administration, Ophthalmic ; Animals ; Cell Line ; Electrophoretic Mobility Shift Assay ; Eye Diseases/genetics ; Eye Diseases/metabolism ; Fluorescent Dyes/chemistry ; Gene Transfer Techniques ; Humans ; Luminescent Measurements ; Mice ; RNA, Small Interfering/administration & dosage ; RNA, Small Interfering/metabolism ; Research Design ; Tissue Distribution ; Workflow
Czasopismo naukowe
Tytuł :
siRNA Design and GalNAc-Empowered Hepatic Targeted Delivery.
Autorzy :
Lu M; School of Life Science, Advanced Research Institute of Multidisciplinary Science, and Institute of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing, China.
Zhang M; School of Life Science, Advanced Research Institute of Multidisciplinary Science, and Institute of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing, China.
Hu B; School of Life Science, Advanced Research Institute of Multidisciplinary Science, and Institute of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing, China.
Huang Y; School of Life Science, Advanced Research Institute of Multidisciplinary Science, and Institute of Engineering Medicine, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing, China. .
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Źródło :
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 77-100.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Gene Transfer Techniques*/adverse effects
RNA Interference*
RNAi Therapeutics*/adverse effects
Acetylgalactosamine/*metabolism
Hepatocytes/*metabolism
Liver/*metabolism
Liver Diseases/*therapy
RNA, Small Interfering/*metabolism
Acetylgalactosamine/toxicity ; Animals ; HEK293 Cells ; Humans ; Liver Diseases/genetics ; Liver Diseases/metabolism ; Mice, Inbred C57BL ; Prealbumin/genetics ; Prealbumin/metabolism ; RNA, Small Interfering/genetics ; RNA, Small Interfering/toxicity ; Research Design ; Toxicity Tests ; Workflow
Czasopismo naukowe
Tytuł :
Cationic Dendrimers for siRNA Delivery: An Overview of Methods for In Vitro/In Vivo Characterization.
Autorzy :
Laurini E; Molecular Biology and Nanotechnology Laboratory (), Department of Engineering and Architecture, University of Trieste, Trieste, Italy. .
Aulic S; Molecular Biology and Nanotechnology Laboratory (), Department of Engineering and Architecture, University of Trieste, Trieste, Italy.
Marson D; Molecular Biology and Nanotechnology Laboratory (), Department of Engineering and Architecture, University of Trieste, Trieste, Italy.
Fermeglia M; Molecular Biology and Nanotechnology Laboratory (), Department of Engineering and Architecture, University of Trieste, Trieste, Italy.
Pricl S; Molecular Biology and Nanotechnology Laboratory (), Department of Engineering and Architecture, University of Trieste, Trieste, Italy.; Department of General Biophysics, Faculty of Biology and Environmental Protection, University of Lodz, Lodz, Poland.
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Źródło :
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 209-244.
Typ publikacji :
Journal Article; Review
MeSH Terms :
RNA Interference*
RNAi Therapeutics*
Transfection*
Dendrimers/*chemistry
RNA, Small Interfering/*genetics
Animals ; Cations ; Cell Line ; Dendrimers/metabolism ; Dendrimers/toxicity ; Humans ; RNA, Small Interfering/chemistry ; RNA, Small Interfering/metabolism
Czasopismo naukowe
Tytuł :
Engaging community pharmacists in quality improvement (QI): a qualitative case study of a partnership between a Higher Education Institute and Local Pharmaceutical Committees.
Autorzy :
Latif A; School of Health Sciences, University of Nottingham, Nottingham, UK .
Gulzar N; School of Pharmacy, De Montfort University, Leicester, UK.
Lowe F; Herefordshire & Worcestershire LPC, Coventry LPC & Warwickshire LPC (Local Pharmaceutical Committee), Evesham, UK.
Ansong T; School of Pharmacy, De Montfort University, Leicester, UK.
Gohil S; School of Pharmacy, De Montfort University, Leicester, UK.
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Źródło :
BMJ open quality [BMJ Open Qual] 2021 Jan; Vol. 10 (1).
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms :
Education, Pharmacy, Continuing*
Pharmacy and Therapeutics Committee*
Program Development*
Quality Improvement*
Schools, Pharmacy*
Community Pharmacy Services/*standards
Pharmacists/*standards
Adult ; Attitude of Health Personnel ; COVID-19 ; Cooperative Behavior ; Curriculum ; Education, Graduate ; Female ; Focus Groups ; Humans ; Male ; Middle Aged ; Motivation ; Pandemics ; Professional Competence ; Professional Role ; Qualitative Research ; Quality of Health Care
Czasopismo naukowe
Tytuł :
siRNA Therapeutics in Ocular Diseases.
Autorzy :
Moreno-Montañés J; Clínica Universidad de Navarra, Pamplona, Spain. .
Bleau AM; Sylentis, Madrid, Spain.
Martínez T; Sylentis, Madrid, Spain.
Vargas B; Sylentis, Madrid, Spain.
González MV; Sylentis, Madrid, Spain.
Jiménez AI; Sylentis, Madrid, Spain.
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Źródło :
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 417-442.
Typ publikacji :
Journal Article; Review
MeSH Terms :
RNA Interference*
RNAi Therapeutics*
Eye/*metabolism
Eye Diseases/*therapy
RNA, Small Interfering/*genetics
Animals ; Biological Availability ; Eye Diseases/genetics ; Eye Diseases/metabolism ; Gene Transfer Techniques ; Humans ; RNA, Small Interfering/metabolism ; Tissue Distribution
Czasopismo naukowe
Tytuł :
Development of siRNA Therapeutics for the Treatment of Liver Diseases.
Autorzy :
Holm A; Center for RNA Medicine, Department of Clinical Medicine, Aalborg University, Copenhagen, Denmark.
Løvendorf MB; Center for RNA Medicine, Department of Clinical Medicine, Aalborg University, Copenhagen, Denmark.
Kauppinen S; Center for RNA Medicine, Department of Clinical Medicine, Aalborg University, Copenhagen, Denmark. .
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Źródło :
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 57-75.
Typ publikacji :
Journal Article; Research Support, Non-U.S. Gov't; Review
MeSH Terms :
RNA Interference*
RNAi Therapeutics*
Liver Diseases/*therapy
RNA, Small Interfering/*therapeutic use
Acetylgalactosamine/analogs & derivatives ; Acetylgalactosamine/therapeutic use ; Amyloid Neuropathies, Familial/genetics ; Amyloid Neuropathies, Familial/metabolism ; Amyloid Neuropathies, Familial/therapy ; Animals ; Gene Transfer Techniques ; Humans ; Liver Diseases/genetics ; Liver Diseases/metabolism ; Porphyrias, Hepatic/diagnosis ; Porphyrias, Hepatic/metabolism ; Porphyrias, Hepatic/therapy ; Pyrrolidines/therapeutic use ; RNA, Small Interfering/genetics ; RNA, Small Interfering/metabolism
SCR Disease Name :
Amyloidosis, Hereditary, Transthyretin-Related
Czasopismo naukowe
Tytuł :
Antitumor effects and mechanisms of CpG ODN combined with attenuated Salmonella-delivered siRNAs against PD-1.
Autorzy :
Jia X; Department of Pathogen Biology, College of Basic Medical Sciences, China Medical University, 77 Puhe Road, Shenyang 110122, PR China.
Guo J; Department of Immunology, Xinxiang Medical University, Xinxiang, Henan 453000, PR China; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China.
Guo S; Department of Immunology, Xinxiang Medical University, Xinxiang, Henan 453000, PR China; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China.
Zhao T; Department of Immunology, Xinxiang Medical University, Xinxiang, Henan 453000, PR China; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China; Xinxiang Key Laboratory of Tumor Vaccine and Immunotherapy, Xinxiang Medical University, 5 Xinxiang, Henan 453000, PR China.
Liu X; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China.
Cheng C; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China.
Wang L; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China.
Zhang B; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China.
Meng C; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China.
Jia H; Institute of Precision Medicine, Xinxiang Medical University, Xinxiang, Henan 453000, PR China; Xinxiang Key Laboratory of Tumor Vaccine and Immunotherapy, Xinxiang Medical University, 5 Xinxiang, Henan 453000, PR China; Department of Pathology, Xinxiang Medical University, Xinxiang, Henan 453000, PR China. Electronic address: .
Luo E; Department of Pathogen Biology, College of Basic Medical Sciences, China Medical University, 77 Puhe Road, Shenyang 110122, PR China. Electronic address: .
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Źródło :
International immunopharmacology [Int Immunopharmacol] 2021 Jan; Vol. 90, pp. 107052. Date of Electronic Publication: 2020 Dec 09.
Typ publikacji :
Journal Article
MeSH Terms :
Genetic Vectors*
RNAi Therapeutics*
Antineoplastic Agents/*pharmacology
Melanoma, Experimental/*therapy
Oligodeoxyribonucleotides/*pharmacology
Programmed Cell Death 1 Receptor/*genetics
RNA, Small Interfering/*genetics
Salmonella/*genetics
Toll-Like Receptor 9/*agonists
Animals ; Cell Line, Tumor ; Combined Modality Therapy ; Cytokines/blood ; Female ; Gene Expression Regulation, Neoplastic ; Male ; Melanoma, Experimental/genetics ; Melanoma, Experimental/immunology ; Melanoma, Experimental/metabolism ; Mice, Inbred C57BL ; Programmed Cell Death 1 Receptor/metabolism ; RNA, Small Interfering/administration & dosage ; RNA, Small Interfering/metabolism ; Time Factors ; Toll-Like Receptor 9/metabolism ; Tumor Burden
Czasopismo naukowe

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