Patent ID: 11931375
Assignee: VOYAGER THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 16:
17. A method for treating and/or ameliorating amyotrophic lateral sclerosis (ALS) in a subject, the method comprising administering to the subject an effective amount of an AAV particle comprising a viral genome and a capsid, wherein said viral genome comprises a modulatory polynucleotide sequence positioned between two inverted terminal repeats (ITRs), wherein said modulatory polynucleotide sequence comprises a nucleic acid sequence encoding a sense strand sequence and an antisense strand sequence of a siRNA duplex, and wherein the sense strand sequence comprises at least 20 consecutive nucleotides from the nucleotide sequence of SEQ ID NO: 2, and the antisense strand sequence comprises at least 20 consecutive nucleotides from the nucleotide sequence of SEQ ID NO: 1, thereby treating and/or ameliorating ALS in the subject.