COMPOSITIONS AND METHODS FOR TREATMENT OF RETINITIS PIGMENTOSA 18 AND RETINITIS PIGMENTOSA 13

摘要

Provided herein are methods and compositions for increasing the expression of PRPF3 or PRPF8, and for treating a subject in need thereof, e.g., a subject with deficient PRPF3 or PRPF8 protein expression or a subject having Retinitis Pigmentosa.