Monitoring biological effects of somatic cell genome editing

Area of Science:

• Biotechnology
• Genetics
• Medical Research

Background:

• CRISPR gene editing therapeutics show great promise but carry risks.
• There's a need for robust preclinical-to-clinical toolkits to ensure safety and efficacy.
• Emerging technologies are crucial for monitoring genome editing effects.

Purpose of the Study:

• To review emerging technologies for monitoring CRISPR genome editing safety and efficacy.
• To highlight tools assessing biological effects across various scales.
• To bridge preclinical development with clinical applications.

Main Methods:

• Direct measurement of DNA editing outcomes (on-target and off-target).
• Utilizing human microphysiological systems (organoids, organs-on-chips) for phenotypic evaluation.
• Employing non-invasive in vivo imaging for biodistribution and activity tracking.

Main Results:

• Technologies assess editing at the DNA, cellular, and whole-organism levels.
• On-target and off-target editing measurements provide functional benchmarks.
• Microphysiological systems allow phenotypic assessment in diverse organ lineages.
• In vivo imaging tracks editor and cell activity and distribution.

Conclusions:

• A suite of complementary technologies monitors genome editors across biological scales.
• These tools are essential for evaluating safety and efficacy in clinical translation.
• Integrated monitoring strategies accelerate the progress of CRISPR therapeutics.