US 11,939,579 B2
Modified U6 promoter system for tissue specific expression
Scott Quenton Harper, Powell, OH (US)
Assigned to RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US)
Filed by RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL, Columbus, OH (US)
Filed on Apr. 26, 2022, as Appl. No. 17/729,751.
Application 17/729,751 is a continuation of application No. 16/089,860, granted, now 11,345,913, previously published as PCT/US2017/025614, filed on Mar. 31, 2017.
Claims priority of provisional application 62/317,524, filed on Apr. 2, 2016.
Prior Publication US 2023/0077409 A1, Mar. 16, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/63 (2006.01); A61K 9/00 (2006.01); A61P 21/00 (2006.01); C07H 21/02 (2006.01); C12N 7/00 (2006.01); C12N 15/113 (2010.01); A61K 48/00 (2006.01); C12Q 1/70 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 9/0019 (2013.01); A61P 21/00 (2018.01); C07H 21/02 (2013.01); C12N 7/00 (2013.01); A61K 48/00 (2013.01); C12N 2310/141 (2013.01); C12N 2320/32 (2013.01); C12N 2330/51 (2013.01); C12N 2710/10043 (2013.01); C12Q 1/70 (2013.01)] 19 Claims
 
1. A method of producing a modified U6 promoter, the method comprising transfecting a host cell with a nucleic acid comprising:
a modified U6 promoter sequence comprising the nucleotide sequence set forth in SEQ ID NO: 4,
a mature guide strand of a miRNA comprising at least one detargeting sequence, wherein the detargeting sequence is a binding site for miRNA-122 and/or miRNA-208, and
5 or 6 thymidines at the 5′ end.