AAV-mediated exon skipping therapy for Usher syndrome, type 2A.
Mauriac SA, Lee J, Zhang J, Jin J, Nist-Lund C, Martin CT, Von Muhlenbrock CR, O'Malley S, Chaves MG, Madison MA, Foster K, Long S, Jacobs C, Kumar SR, Ramirez S, Ichida J, Koehler KR, Holt JR, Géléoc GSG.
Mauriac SA, et al. Among authors: holt jr, geleoc gsg.
Mol Ther. 2026 Jan 7;34(1):179-202. doi: 10.1016/j.ymthe.2025.09.038. Epub 2025 Sep 23.
Mol Ther. 2026.
PMID: 40994011
There are four clinical subtypes, USH1-4, which are associated with mutations in genes important for structure, function, and survival of photoreceptor cells in the retina and sensory hair cells in the inner ear. Genetic mutations in the USH2A gene, which enc …
There are four clinical subtypes, USH1-4, which are associated with mutations in genes important for structure, function, and survival of ph …