US 11,753,641 B2
Structurally designed shRNAs
Gregory J. Hannon, Cold Spring Harbor, NY (US); and Sihem Cheloufi, Boston, MA (US)
Assigned to Cold Spring Harbor Laboratory, Cold Spring Harbor, NY (US)
Filed by Cold Spring Harbor Laboratory
Filed on Feb. 8, 2021, as Appl. No. 17/170,047.
Application 17/170,047 is a continuation of application No. 16/404,271, filed on May 6, 2019, abandoned.
Application 16/404,271 is a continuation of application No. 15/451,016, filed on Mar. 6, 2017, granted, now 10,329,562, issued on Jun. 25, 2019.
Application 15/451,016 is a continuation of application No. 14/630,419, filed on Feb. 24, 2015, granted, now 9,624,494, issued on Mar. 31, 2015.
Application 14/630,419 is a continuation of application No. 13/642,802, granted, now 8,993,532, issued on Mar. 31, 2015, previously published as PCT/US2011/033615, filed on Apr. 22, 2011.
Claims priority of provisional application 61/327,510, filed on Apr. 23, 2010.
Prior Publication US 2021/0254064 A1, Aug. 19, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. C07H 21/02 (2006.01); C07H 21/04 (2006.01); C12N 15/113 (2010.01); C12N 15/11 (2006.01)
CPC C12N 15/113 (2013.01) [C12N 15/111 (2013.01); A01K 2207/05 (2013.01); C12N 2310/14 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2320/30 (2013.01)] 15 Claims
 
1. An adeno-associated virus (AAV) vector comprising a sequence encoding a short hairpin RNA (shRNA) operably linked to an RNA polymerase promoter, said shRNA comprising:
a) a first sequence of 21, 22 or 23 nucleotides complementary to a sequence in a target gene, in a sequence other than the mature sequence of miR-451,
b) a second sequence directly following the first sequence, wherein the entire second sequence is fully complementary to the sequence of the first 17 or 18 nucleotides counted from the 5′ end of the first sequence,
wherein the first sequence and the second sequence form the shRNA, and wherein the last 3 or 4 nucleotides of the first sequence form a loop region in the shRNA.