Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin

Tuberous sclerosis complex (TSC) results from loss of a tumor suppressor gene - TSC1 or TSC2, encoding hamartin and tuberin, respectively. These proteins formed a complex to inhibit mTORC1-mediated cell growth and proliferation. Loss of either protein leads to overgrowth lesions in many vital organs...

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Detalhes bibliográficos
Publicado no:Sci Adv
Main Authors: Cheah, Pike-See, Prabhakar, Shilpa, Yellen, David, Beauchamp, Roberta L., Zhang, Xuan, Kasamatsu, Shingo, Bronson, Roderick T., Thiele, Elizabeth A., Kwiatkowski, David J., Stemmer-Rachamimov, Anat, György, Bence, Ling, King-Hwa, Kaneki, Masao, Tannous, Bakhos A., Ramesh, Vijaya, Maguire, Casey A., Breakefield, Xandra O.
Formato: Artigo
Idioma:Inglês
Publicado em: American Association for the Advancement of Science 2021
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7793581/
https://ncbi.nlm.nih.gov/pubmed/33523984
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.abb1703
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