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AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.
The results demonstrate that the tyrosine-capsid mutant AAV8 (Y733F)vector is effective for treating rapidly degenerating models of retinal degeneration and, moreover, is more therapeutically effective than AAV2 (Y444, 500, 730F) vector with the same promoter-cDNA payload. Expand
Effects of Subretinal Gene Transfer at Different Time Points in a Mouse Model of Retinal Degeneration
It is found that gene therapy was more effective against RD when exposure to light was limited before and after treatment, and long-term preservation of electroretinogram responses, and preservation of retinal structure is observed, indicating that early treatment followed by limited light exposure can improve gene therapy effectiveness for the eyes of rd11 mice. Expand
The Degeneration and Apoptosis Patterns of Cone Photoreceptors in rd11 Mice
The key genes associated with apoptosis indicated that the degeneration and apoptosis of cone photoreceptors began at P14 of rd11 mice, which was a key point for gene therapy. Expand