US 12,215,320 B2
Compositions and methods for gene editing by targeting transferrin
Alan Richard Brooks, Cambridge, MA (US); and Karen Vo, Cambridge, MA (US)
Assigned to CRISPR Therapeutics AG, Zung (CH); and BAYER HEALTHCARE LLC, Whippany, NJ (US)
Appl. No. 16/961,314
Filed by CRISPR Therapeutics AG, Zug (CH); and BAYER HEALTHCARE LLC, Whippany, NJ (US)
PCT Filed Jan. 11, 2019, PCT No. PCT/US2019/013384
§ 371(c)(1), (2) Date Jul. 10, 2020,
PCT Pub. No. WO2019/140330, PCT Pub. Date Jul. 18, 2019.
Claims priority of provisional application 62/617,044, filed on Jan. 12, 2018.
Prior Publication US 2021/0254057 A1, Aug. 19, 2021
Int. Cl. C12N 15/113 (2010.01); A61K 35/407 (2015.01); C12N 9/22 (2006.01); C12N 15/86 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 35/407 (2013.01); C12N 9/22 (2013.01); C12N 15/86 (2013.01); C12N 2510/00 (2013.01); C12N 2750/14143 (2013.01); C12N 2830/42 (2013.01); C12N 2830/50 (2013.01)] 15 Claims
 
1. A system comprising:
a deoxyribonucleic acid (DNA) endonuclease or nucleic acid encoding the DNA endonuclease;
a guide RNA (gRNA) comprising a spacer sequence that is complementary to a sequence within intron 1 of an endogenous transferrin gene in a cell; and
a donor template comprising a nucleic acid sequence encoding a protein-of-interest (POI), wherein the POI is Factor VIII (FVIII).