Abstract
Purpose :
Retinal Degeneration is a group of diseases characterized by rapid degeneration of retinal photoreceptor cells due to genetic or environmental factors. Among them, retinitis pigmentosa is the rapid degeneration of retinal photoreceptor cells due to genetic mutations. With the development of gene therapy and the application of new materials, it has become possible to treat retinitis pigmentosa.
Methods :
RD1 is a mouse model of rapid retinal degeneration due to mutations in the PDE6B gene. We utilized a modified micelle encapsulating rapamycin and adeno-associated virus to express the correct PDE6B gene and injected it into the eyes of mice via subretinal injection.
Results :
We found that the combination of drug and gene therapy can well rescue the photoreceptor cells of RD1 mice and restore their function.
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Conclusions :
This study will provide a good foundation for pre-study at the animal level for the clinical use of gene therapy in combination with advanced materials for drug delivery. It will provide a better treatment for retinal degeneration.
This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.