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Title: ¬¬¬¬CRISPR-derived genome editing therapies: progress from bench to bedside
The development of CRISPR-derived genome editing technologies has enabled the precise manipulation of DNA sequences within the human genome. In this review, we discuss the initial development and cellular mechanism of action of CRISPR nucleases and DNA base editors. We then describe factors that must be taken into consideration when developing these tools into therapeutic agents, including the potential for unintended and off-target edits when using these genome editing tools, and methods to characterize these types of edits. We finish by considering specific challenges associated with bringing a CRISPR-based therapy to the clinic: manufacturing, regulatory oversight and considerations for clinical trials that involve genome editing agents.  more » « less
Award ID(s):
2048207
NSF-PAR ID:
10310630
Author(s) / Creator(s):
; ; ; ;
Date Published:
Journal Name:
Molecular therapy
ISSN:
1525-0024
Format(s):
Medium: X
Sponsoring Org:
National Science Foundation
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