Alnylam Pharmaceuticals, Inc.
siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis

Abstract:

The invention relates to a method of treating ocular amyloidosis by reducing TTR expression in a subject by administering a double-stranded ribonucleic acid (dsRNA) that targets a TTR gene to the retinal pigment epithelium of the subject.