In vivo potency testing of subretinal rAAV5.hCNGB1 gene therapy in the Cngb1 knockout mouse model of retinitis pigmentosa.
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DOI: 10.1089/hum.2021.121
Abstract: Retinitis pigmentosa type 45 (RP45) is an autosomal-recessively inherited blinding disease caused by mutations in the cyclic nucleotide gated channel subunit beta 1 (CNGB1) gene. In this study, we developed and tested a novel gene… read more here.
Keywords: cngb1; therapy; gene; raav5 hcngb1 ... See more keywords