概述:

CRISPR (clustered regularly interspaced short palindromic repeat) and their CRISPR-associated (Cas) proteins constitute the adaptive immune system in bacteria (1-2). This system has been redesigned to create an exemplary genome editing tool for application to RNA-based therapeutics development (3). AsCas12a is a CRISPR-associated endonuclease from Acidaminococcus and it can be engineered to achieve combinatorial genetic screening with high efficiency (2). Engineered AsCas12a further enables accurate editing in hematopoietic stem cells, induced pluripotent stem (iPS) cells, and natural killer (NK) cells and helps fight against sickle cell disease (4).

Genbank编号：

WP_021736722.

参考文献：

1. Horvath, P., et al. CRISPR/Cas, the immune system of bacteria and archaea. Science, 327(5962):167-170.

2. Morange, M. What history tells us XXXVII. CRISPR-Cas: The disconvery of an immune system in prokaryotes. J. Biosci. 40(2):221-223.

3. Gier, R.A., et al. High-performance CRISPR-Cas12a genome editing for combinatorial genetic screening. Nat. Commun. 2020, 11(1):1-9. 4. DeLong, R. Ushering in a new era of RNA-based therapies. Commun Biol. 2021. doi: https://doi.org/10.1038/s42003-021-02150-w