학술논문
Engineered Cas12i2 is a versatile high-efficiency platform for therapeutic genome editing
Document Type
article
Author
Colin McGaw; Anthony J. Garrity; Gabrielle Z. Munoz; Jeffrey R. Haswell; Sejuti Sengupta; Elise Keston-Smith; Pratyusha Hunnewell; Alexa Ornstein; Mishti Bose; Quinton Wessells; Noah Jakimo; Paul Yan; Huaibin Zhang; Lauren E. Alfonse; Roy Ziblat; Jason M. Carte; Wei-Cheng Lu; Derek Cerchione; Brendan Hilbert; Shanmugapriya Sothiselvam; Winston X. Yan; David R. Cheng; David A. Scott; Tia DiTommaso; Shaorong Chong
Source
Nature Communications, Vol 13, Iss 1, Pp 1-11 (2022)
Subject
Language
English
ISSN
2041-1723
Abstract
Cas12i is a genome editing platform with compact size that fits in AAV vector with short 43-mer gRNA, absence of tracrRNA, ability to process pre-crRNA, and high specificity. Here the authors present an unbiased mutational scanning approach to engineer Cas12i, which shows low activity in mammalian cells, and identify single substitutions that significantly improve indel activity.