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

Valérie Matagne(Inserm), Jean‐Christophe Roux(Inserm), Laurent Villard(Inserm), Lydia Saidi(Inserm), Emilie Borloz(Inserm), Yann Ehinger(Inserm)

Neurobiology of Disease

December 28, 2020

10.1016/j.nbd.2020.105235

Cited by 58

Related Papers

Mutations in GRIN2A and GRIN2B encoding regulatory subunits of NMDA receptors cause variable neurodevelopmental phenotypes

|Nature Genetics|2010|509

A non-hallucinogenic psychedelic analogue with therapeutic potential

|Nature|2020|473

Molecular and clinical descriptions of patients with <scp>GABA<sub>A</sub></scp> receptor gene variants (<i><scp>GABRA1</scp>, <scp>GABRB2</scp>, <scp>GABRB3</scp>, <scp>GABRG2</scp></i>): A cohort study, review of literature, and genotype–phenotype correlation

|Epilepsia|2022|80

Epileptic patients with de novo <i><scp>STXBP</scp>1</i> mutations: Key clinical features based on 24 cases

|Epilepsia|2015|67

A codon-optimized Mecp2 transgene corrects breathing deficits and improves survival in a mouse model of Rett syndrome

|Neurobiology of Disease|2016|46