Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1

Tuberous sclerosis complex (TSC) is a tumor suppressor syndrome caused by mutations in TSC1 or TSC2, encoding hamartin and tuberin, respectively. These proteins act as a complex that inhibits mammalian target of rapamycin (mTOR)-mediated cell growth and proliferation. Loss of either protein leads to...

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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Mol Ther Methods Clin Dev
Egile Nagusiak: Prabhakar, Shilpa, Cheah, Pike See, Zhang, Xuan, Zinter, Max, Gianatasio, Maria, Hudry, Eloise, Bronson, Roderick T., Kwiatkowski, David J., Stemmer-Rachamimov, Anat, Maguire, Casey A., Sena-Esteves, Miguel, Tannous, Bakhos A., Breakefield, Xandra O.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: American Society of Gene & Cell Therapy 2019
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC6745533/
https://ncbi.nlm.nih.gov/pubmed/31534984
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.08.003
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