GENE AUGMENTATION THERAPIES FOR INHERITED RETINAL DEGENERATION CAUSED BY MUTATIONS IN THE PRPF31 GENE

Abstract: the present invention relates to methods and compositions for gene therapy of retinitis pigmentosa related to mutations in pre-mrna processing factor 31 (prpf31).

Inventor(s): Eric A. Pierce, Michael H. Farkas, Maria E. Sousa

CPC Classification: A61K48/005 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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