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

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

Rett syndrome (RTT) is a severe X-linked neurodevelopmental disorder that is primarily caused by mutations in the methyl CpG binding protein 2 gene (MECP2). RTT is the second most prevalent genetic cause of intellectual disability in girls, and there is currently no cure for the disease. We have pre...

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Bibliographic Details
Main Authors: Valerie Matagne, Emilie Borloz, Yann Ehinger, Lydia Saidi, Laurent Villard, Jean-Christophe Roux
Format: Article
Language:English
Published: Elsevier 2021-02-01
Series:Neurobiology of Disease
Subjects:
Rett syndrome
Mecp2
AAV9
Gene therapy
Animal models
Side effects
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996120305118

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http://www.sciencedirect.com/science/article/pii/S0969996120305118

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