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Article

Reduced phenotypic severity following adeno-associated virus-mediated Fmr1 gene delivery in fragile X mice.

Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology - 1 Dec 2014

Gholizadeh Shervin, Arsenault Jason, Xuan Ingrid Cong Yang, Pacey Laura K, Hampson David R

Abstract excerpt

Fragile X syndrome (FXS) is a neurodevelopmental disorder caused by a trinucleotide repeat expansion in the FMR1 gene that codes for fragile X mental retardation protein (FMRP). To determine if FMRP expression in the central nervous system could reverse phenotypic deficits in the Fmr1 knockout (KO) mouse model of FXS, we used a single-stranded adeno-associated viral (AAV) vector with viral capsids from serotype 9...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 24998620
DOI: 10.1038/npp.2014.167
PMCID: PMC4229583

Genes

FMR1

Provenance

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