A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model-Reference-Cited by-同舟云学术

A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model

Published:2017-09 Issue:9 Volume:25 Page:2038-2052
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Biferi Maria Grazia,Cohen-Tannoudji Mathilde,Cappelletto Ambra,Giroux Benoit,Roda Marianne,Astord Stéphanie,Marais Thibaut,Bos Corinne,Voit Thomas,Ferry Arnaud,Barkats Martine

Funder

Association Française contre les Myopathies (AFM)

University Pierre et Marie Curie (UPMC)

Institut National de la Santé et de la Recherche Médicale (INSERM)

Centre National de la Recherche Scientifique (CNRS)

Association Institut de Myologie (AIM)

Otto per Mille Waldensian Church

Association pour la Recherche sur la Sclérose Latérale Amyotrophique (ARSLA)

NIHR GOSH Biomedical Research Centre

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference56 articles.

1. Molecular biology of amyotrophic lateral sclerosis: insights from genetics;Pasinelli;Nat. Rev. Neurosci.,2006

2. A controlled trial of riluzole in amyotrophic lateral sclerosis;Bensimon;N. Engl. J. Med.,1994

3. State of play in amyotrophic lateral sclerosis genetics;Renton;Nat. Neurosci.,2014

4. Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis;Rosen;Nature,1993

5. Current developments in gene therapy for amyotrophic lateral sclerosis;Scarrott;Expert Opin. Biol. Ther.,2015

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