US 11,920,148 B2
Compositions and methods for gene editing
Ante Sven Lundberg, Cambridge, MA (US); Samarth Kulkarni, Cambridge, MA (US); Lawrence Klein, Cambridge, MA (US); Hari Kumar Padmanabhan, Cambridge, MA (US); and Yvonne Sarah Aratyn, Cambridge, MA (US)
Assigned to CRISPR THERAPEUTICS AG, Zug (CH)
Appl. No. 16/487,300
Filed by CRISPR THERAPEUTICS AG, Zug (CH)
PCT Filed Feb. 21, 2018, PCT No. PCT/IB2018/000228
§ 371(c)(1), (2) Date Aug. 20, 2019,
PCT Pub. No. WO2018/154387, PCT Pub. Date Aug. 30, 2018.
Claims priority of provisional application 62/587,832, filed on Nov. 17, 2017.
Claims priority of provisional application 62/526,562, filed on Jun. 29, 2017.
Claims priority of provisional application 62/461,829, filed on Feb. 22, 2017.
Prior Publication US 2020/0157565 A1, May 21, 2020
Int. Cl. C12N 15/86 (2006.01); C12N 7/00 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01)
CPC C12N 15/86 (2013.01) [C12N 7/00 (2013.01); C12N 9/22 (2013.01); C12N 15/111 (2013.01); C12N 2310/20 (2017.05); C12N 2750/14143 (2013.01); C12N 2800/80 (2013.01)] 18 Claims
 
1. A method of producing a population of cells comprising a modification in the Angiopoietin-like 3 (ANGPTL3) gene, the method comprising:
introducing into cells (a) a guide RNA (gRNA) gRNA that targets an ANGPTL3 genomic locus or a nucleic acid encoding the gRNA, and (b) a Cas9 endonuclease or a nucleic acid encoding the Cas9 endonuclease; and
producing a population of cells that comprise a modification in the ANGPTL3 gene, wherein the gRNA comprises a spacer sequence that is an RNA sequence having the nucleic acid sequence of SEQ ID NO: 9109, 7168, 7061, 7165, or 9110 in which T is substituted for U, thereby producing the population of cells that comprise a modification in the ANGPTL3 gene.