公开(公告)号：US20240287486A1

公开(公告)日：2024-08-29

申请号：US17921027

申请日：2021-04-21

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： Scot A. Wolfe ,  Kevin Luk ,  PengPeng Liu

IPC: C12N9/22 ,  A61K48/00 ,  C12N15/90

CPC classification number: C12N9/22 ,  A61K48/005 ,  C12N15/907 ,  C07K2319/09 ,  C12N2310/20 ,  C12N2800/80

Abstract: The present invention is related to the field of gene editing. In particular, the present invention is related to the mutation and/or deletion of genetic abnormalities that result in genetic diseases. For example, an improved CRISPR-Cas fusion protein is disclosed where the Cas protein is a Cas12a protein. The Cas12a protein is fused to a variety of nuclear localization signal (NLS) sequences (e.g., c-myc NLS) that are demonstrated to have unexpected and superior gene editing activity when compared to conventional NLS sequences (e.g., SV40 NLS).