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Article

Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model.

Neurobiology of disease - 1 Oct 2015

Prabhakar Shilpa, Zhang Xuan, Goto June, Han Sangyeul, Lai Charles, Bronson Roderick, Sena-Esteves Miguel, Ramesh Vijaya, Stemmer-Rachamimov Anat, Kwiatkowski David J, Breakefield Xandra O

Abstract excerpt

We examined the potential benefit of gene therapy in a mouse model of tuberous sclerosis complex (TSC) in which there is embryonic loss of Tsc1 (hamartin) in brain neurons. An adeno-associated virus (AAV) vector (serotype rh8) expressing a tagged form of hamartin was injected into the cerebral ventricles of newborn pups with the genotype Tsc1(cc) (homozygous for a conditional floxed Tsc1 allele) SynI-cre(+), in...

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Identifiers

PMID: 26019056
DOI: 10.1016/j.nbd.2015.04.018
PMCID: PMC5070799

Genes

TSC1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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