Therapeutic uses of genome editing with CRISPR/Cas systems
申请人:President and Fellows of Harvard College
公开号:US10208319B2
公开(公告)日:2019-02-19
Disclosed herein are methods, compositions, and kits for high efficiency, site-specific genomic editing of cells for treating or preventing genetic blood disorders.
Double stranded mRNA (ds mRNA), e.g., produced in vitro, where one strand encodes a protein of interest and the other strand is hydrogen bonded to at least a portion of the coding region for the protein, as well as methods of making and using the ds mRNA, are provided.