Severe offtarget effects following intravenous delivery of AAV9-MECP2 in a female mouse model of Rett syndrome-Reference-Cited by-同舟云学术

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Severe offtarget effects following intravenous delivery of AAV9-MECP2 in a female mouse model of Rett syndrome

Published:2021-02 Issue: Volume:149 Page:105235
ISSN:0969-9961
Container-title:Neurobiology of Disease
language:en
Short-container-title:Neurobiology of Disease

Author:

Matagne Valerie,Borloz Emilie,Ehinger Yann,Saidi Lydia,Villard Laurent,Roux Jean-Christophe

Funder

INSERM, Aix Marseille University

Association Française du Syndrome de Rett

Publisher

Elsevier BV

Subject

Neurology

Reference54 articles.

1. A segment of the Mecp2 promoter is sufficient to drive expression in neurons;Adachi;Hum. Mol. Genet.,2005

2. Elevated expression of MeCP2 in cardiac and skeletal tissues is detrimental for normal development;Alvarez-Saavedra;Hum. Mol. Genet.,2010

3. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2;Amir;Nat. Genet.,1999

4. Cellular GFP toxicity and immunogenicity: potential confounders in in vivo cell tracking experiments;Ansari;Stem Cell Rev.,2016

5. Production, purification and characterization of adeno-associated vectors;Ayuso;Curr. Gene Ther.,2010

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2. Preclinical Milestones in MECP2 Gene Transfer for Treating Rett Syndrome;Developmental Neuroscience;2024-05-09

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