Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B.
Lau SC, Grati M, Isgrig K, Sinan M, Calabro KR, Zhu J, Ishibashi Y, Ozgur Z, Wafa T, Belyantseva IA, Fitzgerald T, Friedman TB, Boye SL, Boye SE, Chien WW.
Lau SC, et al. Among authors: sinan m.
Mol Ther Methods Clin Dev. 2023 Aug 21;30:534-545. doi: 10.1016/j.omtm.2023.08.012. eCollection 2023 Sep 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 37693946
Free PMC article.
The shaker-1 mouse has a nonsense mutation in Myo7a, is profoundly deaf throughout life, and has significant vestibular dysfunction. Because of the 6.7-kb size of the MYO7A cDNA, a dual-AAV approach was used for gene delivery, which involves splitting human MYO7A cDNA int …
The shaker-1 mouse has a nonsense mutation in Myo7a, is profoundly deaf throughout life, and has significant vestibular dysfunction. …