Methods and compositions for treating diseases, disorders or injury of the CNS

摘要

The present invention relates to non-invasive methods for treating diseases, disorders and injury to the central nervous system (CNS), and in particular to otic compositions and to methods of use thereof.

主权项

1. A method of providing ocular neuroprotection or for preventing, treating or alleviating the effects of, an ocular disease associated with retinal ganglion cell death in a subject, comprising applying to the external ear of the subject an amount of an otic composition comprising:
at least one double-stranded RNA compound which down regulates expression of a target gene in the retina of the subject, wherein expression of said target gene is associated with retinal ganglion cell death; and a penetration enhancer which enhances the penetration of said double-stranded RNA compound through the skin and/or tympanic membrane in the ear of the subject;the amount of the RNA compound in the otic composition being sufficient to reduce the expression of said target gene in the retina, and to inhibit retinal ganglion cell death so as to thereby provide neuroprotection or prevent, treat or alleviate the effects of, said ocular disease.