Related Experiment Video
Updated: Jan 29, 2026
07:56
Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
23.2K
Chronic In Vivo CRISPR-Cas Genome Editing: Challenges, Long-Term Safety, and Outlook
Caroline Bao1, Catherine I Channell1, Yi Hsuan Tseng2
1Department of Ophthalmology, Columbia University Irving Medical Center, New York, NY 10032, USA.
Cells
|January 28, 2026
Summary
This review provides a framework for safe and effective CRISPR gene editing therapies. It guides decisions on transient versus sustained nuclease activity, crucial for developing advanced CRISPR medicines.
Area of Science:
- Molecular Medicine
- Gene Editing Technologies
Background:
- CRISPR/Cas systems offer transformative potential in molecular medicine.
- Current challenges include determining optimal editing duration and ensuring safety during prolonged in vivo delivery.
Purpose of the Study:
- To define conditions for beneficial extended Cas activity while minimizing risks.
- To establish a generalizable framework for safe and effective CRISPR medicines.
Main Methods:
- Examining expression windows and efficacy across different CRISPR-Cas modalities (Cas9, Cas12, Cas13).
- Identifying genome-wide off-target effects and utilizing orthogonal assays.
- Discussing controllable, self-limiting, and recallable editor platforms.
Main Results:
- Distinguishing between durable edits and persistent nuclease exposure is key.
- Validated control levers can enhance safety and efficacy.
- Studies in various models (cell lines to humans) show promising outcomes with manageable safety profiles.
Conclusions:
- A framework for safe, higher-efficacy CRISPR medicines is established.
- Continued monitoring is essential as CRISPR therapies advance.
- Further development is needed for broader clinical applications.
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