US 11,946,064 B2
Compositions and therapeutic methods of microRNA gene delivery
Pierpaolo Peruzzi, Newton, MA (US); and Ennio Antonio Chiocca, Boston, MA (US)
Assigned to The Brigham and Women's Hospital, Inc., Boston, MA (US)
Appl. No. 17/051,527
Filed by The Brigham and Women's Hospital, Inc., Boston, MA (US)
PCT Filed Apr. 30, 2019, PCT No. PCT/US2019/029988
§ 371(c)(1), (2) Date Oct. 29, 2020,
PCT Pub. No. WO2019/213128, PCT Pub. Date Nov. 7, 2019.
Claims priority of provisional application 62/664,362, filed on Apr. 30, 2018.
Prior Publication US 2021/0363542 A1, Nov. 25, 2021
Int. Cl. C12N 15/86 (2006.01); A61K 45/06 (2006.01); A61K 48/00 (2006.01); C12N 15/113 (2010.01); C12Q 1/68 (2018.01)
CPC C12N 15/86 (2013.01) [A61K 45/06 (2013.01); A61K 48/005 (2013.01); C12N 15/113 (2013.01); C12N 2310/11 (2013.01); C12N 2310/141 (2013.01); C12N 2310/16 (2013.01); C12N 2310/531 (2013.01); C12N 2320/30 (2013.01); C12N 2330/50 (2013.01); C12N 2750/14143 (2013.01)] 24 Claims
 
1. A composition comprising a non-naturally occurring microRNA cluster composition, the cluster comprising:
i. a 5′ flanking sequence;
ii. two or more microRNA hairpin domains, wherein the two or more hairpin domains are separated by one or more spacer sequences comprising an aptamer; and
iii. a 3′ flanking sequence.