Current Landscape of Treatments for Wolfram Syndrome.

Animals Clinical Trials, Phase I as Topic Clinical Trials, Phase II as Topic Genetic Therapy / methods Humans Molecular Targeted Therapy Regenerative Medicine / methods Wolfram Syndrome / drug therapy
WFS1 Wolfram syndrome endoplasmic reticulum gene therapy regenerative therapy

Journal

Trends in pharmacological sciences
ISSN: 1873-3735
Titre abrégé: Trends Pharmacol Sci
Pays: England
ID NLM: 7906158

Informations de publication

Date de publication:
10 2019
Historique:
received: 12 04 2019
revised: 18 07 2019
accepted: 18 07 2019
pubmed: 20 8 2019
medline: 7 7 2020
entrez: 18 8 2019
Statut: ppublish

Résumé

Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.

Identifiants

DOI: 10.1016/j.tips.2019.07.011 PMID: 31420094 PMC: PMC7547529
pubmed: 31420094
pii: S0165-6147(19)30163-4
doi: 10.1016/j.tips.2019.07.011
pmc: PMC7547529
mid: NIHMS1634310
pii:
doi:

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

711-714

Subventions

Organisme : NCATS NIH HHS
ID : UH3 TR002065
Pays : United States
Organisme : NIDDK NIH HHS
ID : R21 DK113487
Pays : United States
Organisme : NIDDK NIH HHS
ID : P60 DK020579
Pays : United States
Organisme : NIDDK NIH HHS
ID : P30 DK020579
Pays : United States
Organisme : NIDDK NIH HHS
ID : R01 DK112921
Pays : United States
Organisme : NCATS NIH HHS
ID : UL1 TR002345
Pays : United States
Organisme : NCATS NIH HHS
ID : UH2 TR002065
Pays : United States

Informations de copyright

Copyright © 2019 The Author(s). Published by Elsevier Ltd.. All rights reserved.

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Auteurs

Damien Abreu (D)

Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, St. Louis, MO 63110, USA; Medical Scientist Training Program, Washington University School of Medicine, St. Louis, MO 63110, USA.

Fumihiko Urano (F)

Department of Medicine, Division of Endocrinology, Metabolism, and Lipid Research, Washington University School of Medicine, St. Louis, MO 63110, USA; Department of Pathology and Immunology, Washington University School of Medicine, St. Louis, MO 63110, USA. Electronic address: urano@wustl.edu.

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Current Landscape of Treatments for Wolfram Syndrome.
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Essais cliniques de phase I comme sujet › Current Landscape of Treatments for Wolfram Syndrome.
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Essais cliniques de phase II comme sujet › Current Landscape of Treatments for Wolfram Syndrome.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Current Landscape of Treatments for Wolfram Syndrome.
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Current Landscape of Treatments for Wolfram Syndrome.
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Thérapie moléculaire ciblée › Current Landscape of Treatments for Wolfram Syndrome.
questionsmedicales.fr › Professions de santé › Médecine › Médecine régénérative › Current Landscape of Treatments for Wolfram Syndrome.
questionsmedicales.fr › Maladies endocriniennes › Maladies de l'hypophyse › Diabète insipide › Syndrome de Wolfram › Current Landscape of Treatments for Wolfram Syndrome.