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. |