Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho−/− mouse
作者： Arpad Palfi , Naomi Chadderton , Mary O'Reilly , Kerstin Nagel-Wolfrum , Uwe Wolfrum
DOI: 10.1038/MTM.2015.16
关键词: Transgene 、 Gene delivery 、 Retina 、 Retinal 、 Rhodopsin 、 Cell biology 、 Genetic enhancement 、 Biology 、 Bioinformatics 、 Knockout mouse 、 Retinal degeneration
摘要: As gene therapies for various forms of retinal degeneration progress toward human clinical trial, it will be essential to have a repertoire safe and efficient vectors delivery the target cells. Recombinant adeno-associated virus (AAV) serotype 2/2 has been shown well tolerated in retina provided efficacy patients some inherited degenerations. In this study, AAV2/8 AAV2/rh10 serotypes compared as means mammalian photoreceptor cells using specific promoter transgene expression. Both rescue present rhodopsin knockout mouse, with similar levels benefit evaluated by molecular, histological, functional readouts. Transgene expression were significantly higher (fivefold) 1 week postsubretinal injection when employing AAV2/rh10, indistinguishable 6 weeks postadministration vector. This study reports use provide degenerating provides comparative evaluation respect AAV2/8, regarded providing photoreceptors.
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