	US 12,331,296 B2
	Variant RNAi
Catherine R. O'Riordan, Cambridge, MA (US); Adam Palermo, Cambridge, MA (US); Brenda Richards, Cambridge, MA (US); and Lisa M. Stanek, Cambridge, NJ (US)
Assigned to Genzyme Corporation, Cambridge, MA (US)
Filed by Genzyme Corporation, Cambridge, MA (US)
Filed on Feb. 15, 2023, as Appl. No. 18/169,779.
Application 18/169,779 is a continuation of application No. 16/649,042, granted, now 11,603,529, previously published as PCT/US2018/052221, filed on Sep. 21, 2018.
Claims priority of provisional application 62/561,843, filed on Sep. 22, 2017.
Prior Publication US 2023/0392149 A1, Dec. 7, 2023
Int. Cl. C12N 15/113 (2010.01); A61K 35/761 (2015.01); A61K 48/00 (2006.01); A61P 25/28 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01)

CPC C12N 15/113 (2013.01) [A61K 35/761 (2013.01); A61P 25/28 (2018.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2310/14 (2013.01); C12N 2310/531 (2013.01); C12N 2710/10023 (2013.01); C12N 2710/10043 (2013.01)]

23 Claims

1. A method for treating Huntington's disease in a mammal comprising administering to the mammal an RNAi comprising a first strand comprising a first nucleic acid comprising the sequence 5′-UGGCCGUCCAUCUUGGACCCG-3′ (SEQ ID NO:1) and a second strand comprising a second nucleic acid comprising the sequence 5′-CGGGUCCAAGAUGGACGGCCA-3′ (SEQ ID NO:2) or a first strand comprising a first nucleic acid comprising the sequence 5′-AGUCGGUGUGGUUGACAAGCA-3′ (SEQ ID NO: 7) and a second strand comprising a second nucleic acid comprising the sequence 5′-UGCUUGUCAACCACACCGACU-3′ (SEQ ID NO:8).