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Updated: Mar 25, 2026
08:23
CIRCLE-Seq for Interrogation of Off-Target Gene Editing
Published on: November 1, 2024
1.8K
Genome editing systems in novel therapies
Yoon-Young Jang1, Liuhong Cai2, Zhaohui Ye3
1Department of Oncology and Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21205, USA.
Discovery Medicine
|February 21, 2016
Summary
Modern genome editing technologies like CRISPR offer new therapeutic possibilities by precisely modifying DNA. While promising, challenges in efficiency and safety must be addressed for successful clinical translation.
Area of Science:
- Biotechnology
- Molecular Biology
- Genetics
Background:
- Genome editing enables precise DNA modification.
- Homologous recombination was previously inefficient in human cells.
- Designer endonucleases (ZFN, TALEN, CRISPR) have significantly improved efficiency.
Purpose of the Study:
- To review preclinical and clinical studies of genome editing technologies.
- To discuss challenges and opportunities for translating genome editing into therapies.
Main Methods:
- Review of ZFN, TALEN, and CRISPR systems.
- Analysis of applications in gene and cell therapy.
- Evaluation of preclinical and clinical trial data.
Main Results:
- Genome editing tools offer opportunities for biomedical research and novel therapies.
- Early clinical trials show encouraging results.
- Applications include gene disruption, mutation correction, and gene delivery.
Conclusions:
- Genome editing technologies hold great potential for gene and cell therapies.
- Further research is needed to address off-target effects, preclinical models, and efficiency.
- Increased clinical success is anticipated with technological advancements.
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