Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1.
Prabhakar S, Cheah PS, Zhang X, Zinter M, Gianatasio M, Hudry E, Bronson RT, Kwiatkowski DJ, Stemmer-Rachamimov A, Maguire CA, Sena-Esteves M, Tannous BA, Breakefield XO.
Prabhakar S, et al. Among authors: breakefield xo.
Mol Ther Methods Clin Dev. 2019 Aug 16;15:18-26. doi: 10.1016/j.omtm.2019.08.003. eCollection 2019 Dec 13.
Mol Ther Methods Clin Dev. 2019.
PMID: 31534984
Free PMC article.
In a stochastic mouse model of TSC1 brain lesions, complete loss of Tsc1 is achieved in homozygous Tsc1-floxed mice in a subpopulation of neural cells in the brain by intracerebroventricular (i.c.v.) injection at birth of an adeno-associated virus (AAV) vector encoding Cre …
In a stochastic mouse model of TSC1 brain lesions, complete loss of Tsc1 is achieved in homozygous Tsc1-floxed mice in a subpopulation of ne …