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Updated: Oct 17, 2025

Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
CRISPR-derived genome editing therapies: Progress from bench to bedside
Holly A Rees1, Alex C Minella1, Cameron A Burnett2
1Beam Therapeutics, Cambridge, MA, USA.
Abstract:
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, including manufacturing, regulatory oversight, and considerations for clinical trials that involve genome editing agents.
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