AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy-Reference-Cited by-同舟云学术

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AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy

Published:2013-08 Issue:8 Volume:21 Page:1551-1558
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Le Hir Maëva,Goyenvalle Aurélie,Peccate Cécile,Précigout Guillaume,Davies Kay E,Voit Thomas,Garcia Luis,Lorain Stéphanie

Publisher

Elsevier BV

Subject

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

Reference30 articles.

1. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy;Harper;Nat Med,2002

2. Systemic administration of PRO051 in Duchenne's muscular dystrophy;Goemans;N Engl J Med,2011

3. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study;Cirak;Lancet,2011

4. U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping;Brun;Cell Mol Life Sci,2003

5. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping;Goyenvalle;Science,2004

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2. Therapeutic approaches for Duchenne muscular dystrophy;Nature Reviews Drug Discovery;2023-08-31

3. Production of Duchenne muscular dystrophy cellular model using CRISPR-Cas9 exon deletion strategy;Molecular and Cellular Biochemistry;2023-06-08

4. Lifelong Outcomes of Systemic Adeno-Associated Virus Micro-Dystrophin Gene Therapy in a Murine Duchenne Muscular Dystrophy Model;Human Gene Therapy;2023-05-02

5. Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle;Proceedings of the National Academy of Sciences;2023-01-03

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