The Broad Institute, Inc.;The Brigham and Women's Hospital, Inc.
发明人:
申请号:
EP18726260.5
公开号:
EP3612232A1
申请日:
2018.04.20
申请国别(地区):
EP
年份:
2020
代理人:
摘要:
The disclosure includes zinc prodrugs for targeted delivery of therapeutic, diagnostic or imaging agents to β-cells and methods of use therefor. The disclosure also includes targeted delivery of small molecules to β-cells that stabilize and activate CRISPR effector proteins comprising at least one destabilization domain, to enable CRISPR-based genome editing and transcriptional activation or repression in β-cells.