METHODS OF RESTORING LYSOSOMAL FUNCTION OF RETINAL PIGMENT EPITHELIAL CELLS BY ACTIVATION OF TFEB

摘要

A method of restoring lysosomal function of retinal pigment epithelial (RPE) cells and a method of preventing and/or treating age-related macular degeneration (AMD), Stargardt's macular retinal degeneration, neurodegenerative disease, or diabetic retinopathy in a subject are provided. The methods comprise administering (i) a nucleic acid encoding a polypeptide comprising a constitutively active form of transcription factor EB (TFEB) or (ii) the polypeptide to a subject in need thereof. Associated polypeptides, nucleic acids, vectors, and compositions thereof also are provided.