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Bioconjugate chemistry

In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges.


PMID 28263568

Abstract

The successful use of clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9-based gene editing for therapeutics requires efficient in vivo delivery of the CRISPR components. There are, however, major challenges on the delivery front. In this Topical Review, we will highlight recent developments in CRISPR delivery, and we will present hurdles that still need to be overcome to achieve effective in vivo editing.