Patent ID: 11896669
Assignee: UNIVERSITY OF MASSACHUSETTS
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 18:
19. A method of delivering an siRNA molecule to the central nervous system of a subject, the method comprising intrastriatally, intrathecally, or intracerebroventricularly administering to the subject a di-branched oligonucleotide compound comprising two siRNA molecules covalently bound to one another by way of a linker,
wherein:
(a) each siRNA molecule of the di-branched oligonucleotide compound comprises an antisense strand having complementarity to a target gene and a sense strand having complementarity to the antisense strand;
(b) from 80% to 100% of the nucleotides in each siRNA molecule of the di-branched oligonucleotide compound are chemically modified; and
(c) each antisense strand and each sense strand are, independently, from 15 to 30 nucleotides in length; and
(d) wherein the 5′ end of each antisense strand comprises an (E)-vinylphosphonate terminal group.