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Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B

Usher syndrome is the most common cause of deafness-blindness in the world. Usher syndrome type 1B (USH1B) is associated with mutations in MYO7A. Patients with USH1B experience deafness, blindness, and vestibular dysfunction. In this study, we applied adeno-associated virus (AAV)-mediated gene thera...

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Detalles Bibliográficos
Autores principales: Lau, Samantha C., Grati, Mhamed, Isgrig, Kevin, Sinan, Moaz, Calabro, Kaitlyn R., Zhu, Jianliang, Ishibashi, Yasuko, Ozgur, Zeynep, Wafa, Talah, Belyantseva, Inna A., Fitzgerald, Tracy, Friedman, Thomas B., Boye, Sanford L., Boye, Shannon E., Chien, Wade W.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10491803/
https://www.ncbi.nlm.nih.gov/pubmed/37693946
http://dx.doi.org/10.1016/j.omtm.2023.08.012