rAAV-VECTORED RIBOZYME COMPOSITIONS AND METHODS FOR THE TREATMENT OF RETINAL DISEASES

摘要

Disclosed are ribozymes, as well as compositions, vectors, virus particles, host cells, and therapeutic kits that comprise them, which have been shown to be useful in the manufacture of medicaments and in the treatment of diseases of the mammalian eye, and in particular retinopathy and macular degeneration. Also disclosed are methods and uses of these compositions in the reduction, elimination, and/or amelioration of symptoms of diseases of the human eye, including for example, loss of vision, retinitis, retinopathy, and blindness.