CRISPR-based gene editing is presently being tried in many clinical trials

Abstract

CRISPR is a bacterial host defense system that may work as "molecular scissors" in eukaryotic cells to permanently modify genetic coding. Some barriers to using CRISPR as a therapeutic include guaranteeing adequate delivery of the RNP complex to the proper cell/tissue and showing safe and effective editing. Off-target editing (i.e., unwanted modification in a non-target DNA location) may result in a range of safety problems impacting normal cell function. The degree of cell editing events, including off-target modifications, is known to be altered by in vitro dosage and time of exposure to active RNP complexes. The safety of these drugs relies heavily on preventing unwanted mutations, off-target mutations, and any genomic rearrangements, all of which may have harmful implications.