US 12,110,499 B2
Homology directed repair compositions for the treatment of hemoglobinopathies
Andrew Scharenberg, Seattle, WA (US); Kyle Jacoby, Seattle, WA (US); Hans-Peter Kiem, Seattle, WA (US); David J. Rawlings, Seattle, WA (US); Christopher Lux, Bremerton, WA (US); Sowmya Pattabhi, Bellevue, WA (US); and Olivier M. Humbert, Seattle, WA (US)
Assigned to Seattle Children's Hospital, Seattle, WA (US)
Appl. No. 16/608,182
Filed by Seattle Children's Hospital, Seattle, WA (US); and Fred Hutchinson Cancer Center, Seattle, WA (US)
PCT Filed Apr. 24, 2018, PCT No. PCT/US2018/029235
§ 371(c)(1), (2) Date Oct. 24, 2019,
PCT Pub. No. WO2018/200597, PCT Pub. Date Nov. 1, 2018.
Claims priority of provisional application 62/488,927, filed on Apr. 24, 2017.
Prior Publication US 2021/0222201 A1, Jul. 22, 2021
Int. Cl. C12N 15/90 (2006.01); A61K 35/28 (2015.01); A61P 9/00 (2006.01); C07K 14/805 (2006.01); C12N 15/11 (2006.01); C12N 15/86 (2006.01)
CPC C12N 15/907 (2013.01) [A61K 35/28 (2013.01); A61P 9/00 (2018.01); C07K 14/805 (2013.01); C12N 15/11 (2013.01); C12N 15/86 (2013.01); C12N 2310/20 (2017.05); C12N 2750/14143 (2013.01); C12N 2800/80 (2013.01)] 18 Claims
 
1. A method for increasing gamma globin expression in a hematopoietic stem or progenitor cell comprising introducing into the cell one or more engineered nucleases that cleave a target site set forth in SEQ ID NO: 9 and
a DNA donor repair template having the sequence as set forth in SEQ ID NOs: 17, 18, 19, 20, or 21,
whereby the DNA donor repair template is inserted into the cell genome by homology directed repair at a double strand break introduced by the one or more engineered nucleases.