Scholarlink

Background <p>As the ancestor of CRISPR-Cas12 nucleases, TnpB represents the most compact gene editing tool currently available. Recent studies have identified multiple TnpB systems with gene editing activity in mammalian cells, and the potential of TnpB in treating diseases has been demonstrated in animal models. However, the editing characteristics of various TnpB systems, comparable to CRISPR tools, require more extensive investigation.</p> Results <p>Using a standardized evaluation framework, we conduct a thorough analysis of the editing properties of four TnpB variants alongside representative Cas12 and Cas9 tools applications. Overall, TnpBs exhibit intermediate editing activity and safety profiles among all tested systems, with <i>IS</i>Ymu1 TnpB demonstrating a good performance in both editing activity and specificity. Considering its compact size, potent editing efficiency and high specificity, <i>IS</i>Ymu1 TnpB represents a promising candidate for gene therapy.</p> Conclusions <p>By comprehensively analyzing genome editing outcomes, we characterize TnpB systems for genome editing and identify <i>IS</i>Ymu1 TnpB as an optimal miniature RNA-guided genome editors with balanced performance, highlighting its potential for therapeutic applications.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Comprehensive assessment of activity, specificity, and safety of hypercompact TnpB systems for gene editing

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年1月21日

查看全文
  • Changchang Xin,
  • Guanghai Xiang,
  • Shiwei Cao,
  • Yuhong Wang,
  • Shaopeng Yuan,
  • Xinyi Liu,
  • Yongyuan Huo,
  • Jing Sun,
  • Xichen Wan,
  • Duan Liu,
  • Jiaxu Hong,
  • Jiazhi Hu,
  • Haoyi Wang

摘要

Background

As the ancestor of CRISPR-Cas12 nucleases, TnpB represents the most compact gene editing tool currently available. Recent studies have identified multiple TnpB systems with gene editing activity in mammalian cells, and the potential of TnpB in treating diseases has been demonstrated in animal models. However, the editing characteristics of various TnpB systems, comparable to CRISPR tools, require more extensive investigation.

Results

Using a standardized evaluation framework, we conduct a thorough analysis of the editing properties of four TnpB variants alongside representative Cas12 and Cas9 tools applications. Overall, TnpBs exhibit intermediate editing activity and safety profiles among all tested systems, with ISYmu1 TnpB demonstrating a good performance in both editing activity and specificity. Considering its compact size, potent editing efficiency and high specificity, ISYmu1 TnpB represents a promising candidate for gene therapy.

Conclusions

By comprehensively analyzing genome editing outcomes, we characterize TnpB systems for genome editing and identify ISYmu1 TnpB as an optimal miniature RNA-guided genome editors with balanced performance, highlighting its potential for therapeutic applications.