Ontology highlight
ABSTRACT:
SUBMITTER: Prabhakar S
PROVIDER: S-EPMC5070799 | biostudies-literature | 2015 Oct
REPOSITORIES: biostudies-literature
Prabhakar Shilpa S Zhang Xuan X Goto June J Han Sangyeul S Lai Charles C Bronson Roderick R Sena-Esteves Miguel M Ramesh Vijaya V Stemmer-Rachamimov Anat A Kwiatkowski David J DJ Breakefield Xandra O XO
Neurobiology of disease 20150524
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 which Tsc1 is lost selectively in neurons starting at embryonic day 12. Vector-treate ...[more]