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Dual AAV based PCDH15 gene therapy achieves sustained rescue of visual function in a mouse model of Usher syndrome 1F

Dual AAV based PCDH15 gene therapy achieves sustained rescue of visual function in a mouse model of Usher syndrome 1F.

Riaz S, Sethna S, Duncan T, Naeem MA, Redmond TM, Riazuddin S, Riazuddin S, Carvalho LS, Ahmed ZM.

Mol Ther. 2023 Oct 20:S1525-0016(23)00558-0. doi: 10.1016/j.ymthe.2023.10.017. Online ahead of print.

PMID: 37864333

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