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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6745533/
https://www.ncbi.nlm.nih.gov/pubmed/31534984
http://dx.doi.org/10.1016/j.omtm.2019.08.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6745533/
https://www.ncbi.nlm.nih.gov/pubmed/31534984
http://dx.doi.org/10.1016/j.omtm.2019.08.003

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