	US 12,296,026 B2
	Transcription regulatory elements and uses thereof
John T. Gray, San Francisco, CA (US)
Assigned to Astellas Gene Therapies, Inc., South San Francisco, CA (US)
Appl. No. 16/966,521
Filed by Audentes Therapeutics, Inc., San Francisco, CA (US)
PCT Filed Feb. 5, 2019, PCT No. PCT/US2019/016692 § 371(c)(1), (2) Date Jul. 31, 2020, PCT Pub. No. WO2019/153009, PCT Pub. Date Aug. 8, 2019.
Claims priority of provisional application 62/626,561, filed on Feb. 5, 2018.
Prior Publication US 2021/0162073 A1, Jun. 3, 2021
Int. Cl. A61K 48/00 (2006.01); A61P 3/00 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01)

CPC A61K 48/0058 (2013.01) [A61P 3/00 (2018.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/008 (2013.01)]

11 Claims

1. A nucleic acid regulatory element comprising:

(i) a first segment;

(ii) a second segment; and

(iii) a third segment;

wherein the components are operably linked to each other in a 5′-to-3′ direction as:

first segment-second segment-third segment;

and wherein:

the first segment comprises a synapsin promoter having the nucleic acid sequence of SEQ ID NO: 8,

the second segment comprises an apolipoprotein E hepatic control region (ApoE-HCR) having the nucleic acid sequence of SEQ ID NO: 1, and

the third segment comprises a desmin promoter having the nucleic acid sequence of SEQ ID NO: 7.