US 11,701,390 B2
Gene therapy
Brian Bigger, Manchester (GB); and Hélène Gleitz, Rotterdam (NL)
Assigned to The University of Manchester, Manchester (GB)
Appl. No. 16/484,311
Filed by The University of Manchester, Manchester (GB)
PCT Filed Feb. 7, 2018, PCT No. PCT/GB2018/050347
§ 371(c)(1), (2) Date Aug. 7, 2019,
PCT Pub. No. WO2018/146473, PCT Pub. Date Aug. 16, 2018.
Claims priority of application No. 1701968 (GB), filed on Feb. 7, 2017.
Prior Publication US 2020/0000854 A1, Jan. 2, 2020
Int. Cl. C12N 15/63 (2006.01); C07H 21/04 (2006.01); A61K 35/28 (2015.01); C07K 14/775 (2006.01); C12N 5/16 (2006.01); C12N 15/79 (2006.01)
CPC A61K 35/28 (2013.01) [C07K 14/775 (2013.01); C12N 5/16 (2013.01); C12N 15/63 (2013.01); C07H 21/04 (2013.01); C12N 15/79 (2013.01); C12N 2510/00 (2013.01)] 7 Claims
 
1. A nucleic acid comprising an iduronate-2-sulfatase (IDS) gene sequence and a tandem repeat of an Apolipoprotein E (ApoEII) gene sequence, further comprising an intervening linker sequence located between the IDS sequence and the ApoEII sequence, wherein the intervening linker sequence comprises the sequence according to SEQ ID No. 4 or a derivative sequence having at least 95% homology to SEQ ID No. 4.