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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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Bibliographic Details
Main Authors: Prabhakar, Shilpa, Cheah, Pike See, Zhang, Xuan, Zinter, Max, Gianatasio, Maria, Hudry, Eloise, Bronson, Roderick T, Kwiatkowski, David J, Rachamimov, Anat Stemmer, Maguire, Casey A, Sena-Esteves, Miguel, Tannous, Bakhos A, Breakefield, Xandra O
Format: Article
Language:English
Published: Cell Press 2019
Online Access:http://psasir.upm.edu.my/id/eprint/81248/1/Long-term%20therapeutic%20efficacy%20of%20intravenous%20AAV.pdf
http://psasir.upm.edu.my/id/eprint/81248/
https://pubmed.ncbi.nlm.nih.gov/31534984/
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