US 12,331,086 B2
Artificially engineered SC function control system
Seok Joong Kim, Seoul (KR); Dong Woo Song, Seoul (KR); Young Bin Hong, Seoul (KR); Byung Ok Choi, Seoul (KR); Jae Young Lee, Seoul (KR); and Jung Min Lee, Seoul (KR)
Assigned to TOOLGEN INCORPORATED, Seoul (KR)
Appl. No. 16/349,672
Filed by TOOLGEN INCORPORATED, Seoul (KR)
PCT Filed Sep. 28, 2017, PCT No. PCT/KR2017/010897
§ 371(c)(1), (2) Date May 14, 2019,
PCT Pub. No. WO2018/088694, PCT Pub. Date May 17, 2018.
Claims priority of provisional application 62/421,516, filed on Nov. 14, 2016.
Prior Publication US 2019/0276503 A1, Sep. 12, 2019
Int. Cl. C12N 15/113 (2010.01); A61K 35/30 (2015.01); C07K 14/315 (2006.01); C12N 15/86 (2006.01)
CPC C07K 14/315 (2013.01) [A61K 35/30 (2013.01); C12N 15/113 (2013.01); C12N 15/86 (2013.01); C12N 2310/10 (2013.01); C12N 2310/20 (2017.05)] 5 Claims
 
1. A composition for editing a PMP22 gene in a cell, comprising:
a Cas9 protein derived from Streptococcus pyogenes or a nucleic acid encoding the Cas9 protein; and
a guide RNA comprising a crRNA and a tracrRNA or a nucleic acid encoding the guide RNA,
wherein the guide RNA targets a sequence of SEQ ID NOs: 14 to 17, which is a TATA-box site of a human PMP22 gene.