Patent ID: 9701984
Date: 2017-07-11
CPC Classifications: A61K,A61P,C12N,C12Y

Claim:
1. A method for delivering a transgene to CNS tissue in a subject, the method comprising: administering an effective amount of a rAAV comprising a promoter operably linked with a transgene to central nervous system (CNS) tissue by intrathecal administration, wherein the rAAV infects cells of the CNS of the subject, wherein the transgene encodes an inhibitory RNA that has the sequence of SEQ ID NO: 26, wherein the inhibitory RNA is complementary with and specifically binds to a target site sequence of the SOD1 mRNA and inhibits expression of SOD1 protein in the subject, wherein the target site sequence of the SOD1 mRNA comprises at least 5 nucleotides that are complementary with a sequence as set forth in SEQ ID NO: 26.