	US 12,214,056 B2
	Therapeutic applications of CPF1-based genome editing
Charles A. Gersbach, Durham, NC (US); Sarina Madhavan, Katy, TX (US); and Christopher Nelson, Durham, NC (US)
Assigned to Duke University, Durham, NC (US)
Appl. No. 16/318,745
Filed by Duke University, Durham, NC (US)
PCT Filed Jul. 19, 2017, PCT No. PCT/US2017/042921 § 371(c)(1), (2) Date Jan. 18, 2019, PCT Pub. No. WO2018/017754, PCT Pub. Date Jan. 25, 2018.
Claims priority of provisional application 62/363,888, filed on Jul. 19, 2016.
Prior Publication US 2019/0151476 A1, May 23, 2019
Int. Cl. A61K 48/00 (2006.01); A61K 31/7105 (2006.01); A61K 38/46 (2006.01); A61P 21/00 (2006.01); C07K 14/47 (2006.01); C12N 9/22 (2006.01); C12N 15/11 (2006.01); C12N 15/113 (2010.01); C12N 15/90 (2006.01)

CPC A61K 48/0066 (2013.01) [A61K 31/7105 (2013.01); A61K 38/465 (2013.01); A61K 48/0008 (2013.01); A61K 48/0058 (2013.01); A61K 48/0075 (2013.01); A61K 48/0091 (2013.01); A61P 21/00 (2018.01); C07K 14/4708 (2013.01); C12N 9/22 (2013.01); C12N 15/111 (2013.01); C12N 15/113 (2013.01); C12N 15/907 (2013.01); C12N 2310/20 (2017.05); C12N 2320/33 (2013.01); C12N 2750/14143 (2013.01); Y02A 50/30 (2018.01)]

12 Claims

1. A DNA targeting composition comprising a first Cpf1 gRNA and a second Cpf1 gRNA selected from the group consisting of:

(i) a first Cpf1 gRNA comprising a polynucleotide encoded by the sequence of SEQ ID NO: 54 and a second Cpf1 gRNA comprising a polynucleotide encoded by the sequence of SEQ ID NO: 62;

(ii) a first Cpf1 gRNA comprising a polynucleotide encoded by the sequence of SEQ ID NO: 55 and a second Cpf1 gRNA comprising a polynucleotide encoded by the sequence of SEQ ID NO: 63; and

(iii) a first Cpf1 gRNA comprising a polynucleotide encoded by the sequence of SEQ ID NO: 56 and a second Cpf1 gRNA comprising a polynucleotide encoded by the sequence of SEQ ID NO: 61.