US 12,319,924 B2
Liver-specific viral promoters and methods of using the same
Jacek Lubelski, Amsterdam (NL); David Johannes Francois Du Plessis, Amsterdam (NL); Ying Pui Liu, Amsterdam (NL); Olivier Ter Brake, Amsterdam (NL); Juan Manuel Iglesias Gonzalez, Lothian (GB); Ross Fraser, Lothian (GB); and Michael Roberts, Lothian (GB)
Assigned to uniQure IP B.V., Amsterdam (NL)
Filed by uniQure IP B.V., Amsterdam (NL)
Filed on May 14, 2021, as Appl. No. 17/320,758.
Application 17/320,758 is a continuation of application No. PCT/EP2019/081743, filed on Nov. 19, 2019.
Claims priority of application No. 18207027 (EP), filed on Nov. 19, 2018.
Prior Publication US 2022/0073943 A1, Mar. 10, 2022
Int. Cl. C12N 15/85 (2006.01); A61P 1/16 (2006.01); C12N 15/86 (2006.01); A61K 48/00 (2006.01)
CPC C12N 15/85 (2013.01) [A61P 1/16 (2018.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2750/14123 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01); C12N 2830/008 (2013.01)] 22 Claims
 
1. A synthetic polynucleotide, comprising:
(a) HNF1/HNF3 (SEQ ID NO:1),
(b) HNF3/HNF3 (SEQ ID NO:2),
(c) c/EBP/HNF4 (SEQ ID NO:3),
(d) HS_CRM2/HNF3 (SEQ ID NO:4), and
(e) HS_CRM8 (SEQ ID NO:6) or a variant thereof;
or wherein the variant of HS_CRM8 is selected from the group consisting of SEQ ID NO:5, SEQ ID NO:87, SEQ ID NO:88, SEQ ID NO:89, SEQ ID NO:90, SEQ ID NO:91, SEQ ID NO:92, SEQ ID NO:93, SEQ ID NO:94, SEQ ID NO:95, SEQ ID NO:96, SEQ ID NO:97, SEQ ID NO:98, SEQ ID NO:99, and SEQ ID NO:100.