Related Experiment Video
Updated: Oct 8, 2025
08:23
CIRCLE-Seq for Interrogation of Off-Target Gene Editing
Published on: November 1, 2024
940
Therapeutic application of sequence-specific binding molecules for novel genome editing tools
Juki Nakao1, Tsuyoshi Yamamoto1, Asako Yamayoshi2
1Chemist. of Funct. Mol., Grad. Sch. Biomed. Sci., Nagasaki Univ, Japan.
Drug Metabolism and Pharmacokinetics
|January 2, 2022
Summary
Genome editing, including CRISPR-Cas9, offers new disease treatments but faces clinical limitations. Next-generation technologies and multidisciplinary approaches are crucial for advancing gene therapy development.
Area of Science:
- Biotechnology
- Genetics
- Molecular Biology
Background:
- Genome editing tools promise novel treatments for diseases.
- The CRISPR-Cas9 system significantly advanced gene-editing capabilities.
- Current gene-editing tools face limitations hindering clinical application.
Purpose of the Study:
- To review the history, types, and limitations of genome editing tools.
- To explore next-generation technologies addressing current gene therapy challenges.
- To provide a foundation for the clinical development of genome editing.
Main Methods:
- Literature review of genome editing technologies.
- Analysis of limitations in current gene therapy protocols.
- Examination of emerging technologies for overcoming these limitations.
Main Results:
- The development of genome editing has expanded treatment possibilities.
- CRISPR-Cas9 is a key but not universally applicable gene-editing tool.
- Next-generation technologies show promise in overcoming existing hurdles.
Conclusions:
- Genome editing holds significant therapeutic potential but requires further development.
- Addressing limitations necessitates innovative approaches and multidisciplinary collaboration.
- Accelerating clinical translation of gene editing requires overcoming current bottlenecks.
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