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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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Principais autores: Samantha C. Lau, Mhamed Grati, Kevin Isgrig, Moaz Sinan, Kaitlyn R. Calabro, Jianliang Zhu, Yasuko Ishibashi, Zeynep Ozgur, Talah Wafa, Inna A. Belyantseva, Tracy Fitzgerald, Thomas B. Friedman, Sanford L. Boye, Shannon E. Boye, Wade W. Chien
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2023-09-01
Colecção:Molecular Therapy: Methods & Clinical Development
Assuntos:
gene therapy
hearing loss
Usher syndrome
USH1B
MYO7A
AAV
Acesso em linha:http://www.sciencedirect.com/science/article/pii/S2329050123001262
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