CRISPR Therapeutics AG
COMPOSITIONS AND METHODS FOR DELIVERING TRANSGENES

Abstract:

Provided herein are compositions, methods, and systems for targeted delivery of nucleic acids, including DNA and RNA, to a target cell. Also provided are compositions, methods, and systems for expressing a transgene in a cell by genomic editing. Further provided are compositions, methods, and systems for knocking in a gene-of-interest (GOI) into a target genomic locus in the genome, in particular the locus of albumin gene. Also provided are compositions, methods, and systems for treating a subject having or suspected of having a disorder or health condition employing ex vivo and/or in vivo genome editing.