US 9,809,817 B2
Oligonucleotide compounds for targeting huntingtin mRNA
Anastasia Khvorova, Westborough, MA (US); Neil Aronin, Newtonville, MA (US); and Julia Alterman, Worcester, MA (US)
Assigned to UNIVERSITY OF MASSACHUSETTS, Boston, MA (US)
Filed by University of Massachusetts, Boston, MA (US)
Filed on Apr. 1, 2016, as Appl. No. 15/89,319.
Claims priority of provisional application 62/289,274, filed on Jan. 31, 2016.
Claims priority of provisional application 62/142,731, filed on Apr. 3, 2015.
Prior Publication US 2016/0355808 A1, Dec. 8, 2016
Int. Cl. C12N 15/113 (2010.01); A61K 31/713 (2006.01); A61K 9/00 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 9/0085 (2013.01); A61K 31/713 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/343 (2013.01); C12N 2310/344 (2013.01); C12N 2310/346 (2013.01); C12N 2310/3515 (2013.01); C12N 2310/3517 (2013.01); C12N 2310/3519 (2013.01); C12N 2310/52 (2013.01); C12N 2320/11 (2013.01); C12N 2320/30 (2013.01); C12N 2320/32 (2013.01); C12N 2320/51 (2013.01)] 41 Claims
 
1. A vector for inhibiting the expression of HTT gene in a cell, said vector comprising a regulatory sequence operably linked to a nucleotide sequence that encodes a dsRNA molecule substantially complementary to 5′ CAGUAAAGAGAUUAA 3′ (SEQ ID NO:1), 5′ AUAUCAGUAAAGAGA 3′ (SEQ ID NO:2) or 5′ CUCAGGAUUUAAAAU 3′ (SEQ ID NO:3), wherein said dsRNA molecule is between 10 and 35 bases in length.