THE BROAD INSTITUTE, INC.;MASSACHUSETTS INSTITUTE OF TECHNOLOGY
发明人:
Feng ZHANG,David Arthur SCOTT,Winston Xia YAN,Sourav CHOUDHURY,Matthias HEIDENREICH
申请号：
US16325898
公开号：
US20200080112A1
申请日:
2017.08.17
申请国别(地区):
US
年份:
2020
代理人:
摘要：
Embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type V effector protein. In certain other example embodiments, the Type V effector protein is Cpf1. Embodiments disclosed herein are directed to viral vectors for delivery of CRISPR-Cas effector proteins, including Cpf1. In certain example embodiments, the vectors are designed so as to allow packaging of the CRISPR-Cas effector protein within a single vector. There is also an increased interest in the design of compact promoters for packing and thus expressing larger transgenes for targeted delivery and tissue-specificity. Thus, in another aspect certain embodiments disclosed herein are directed to delivery vectors, constructs, and methods of delivering larger genes for systemic delivery.
