Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin.
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Cheah PS, et al. Among authors: ramesh v.
Sci Adv. 2021 Jan 8;7(2):eabb1703. doi: 10.1126/sciadv.abb1703. Print 2021 Jan.
Sci Adv. 2021.
PMID: 33523984
Free PMC article.