Scholarlink

<p>CRISPR-based genome editing therapeutics are entering the clinic, offering transformative potential but also presenting potential risks. Preclinical-to-clinical toolkits are needed to assess the safety and efficacy of these new therapies and accelerate progress. Emerging technologies to monitor the biological effects of genome editors cover a range of biological scales, from the direct measurement of editing outcomes in DNA, to human microphysiological systems, and non-invasive&#xa0;in vivo imaging. Measurements of on-target and off-target editing outcomes, including sequences unique to humans, provide essential benchmarks to understand functional responses. Microphysiological systems, including organoids and organs-on-chips, enable phenotypic evaluations of editing strategies in varied organ lineages and disease states. Non-invasive imaging modalities can track the biodistribution and activities of genome editors and edited cells in vivo. Collectively, these technologies provide complementary insights across different scales, from the single nucleotide to the whole organism, bridging preclinical therapeutics development with clinical trials.</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

Monitoring biological effects of somatic cell genome editing

  • 期刊论文

  • 发表日期：

    2026年1月13日

查看全文
  • Benjamin S. Freedman,
  • Jeff W. M. Bulte,
  • Bruce R. Conklin,
  • Luke M. Judge,
  • Melinda R. Dwinell,
  • Aron M. Geurts,
  • Madeleine J. Sitton,
  • Vineet Mahajan,
  • Samira Kiani,
  • Charles A. Gersbach,
  • Mo R. Ebrahimkhani,
  • John J. Kelly,
  • John A. Ronald,
  • Ryuji Morizane,
  • Navin Gupta,
  • Ali Shakeri-Zadeh,
  • Nicole Vo,
  • Krishanu Saha,
  • Shivani Saxena,
  • David M. Gamm,
  • Divya Sinha,
  • Alice F. Tarantal,
  • Moriel Vandsburger,
  • Azusa Matsubara,
  • Hongxia Fu,
  • Shengdar Q. Tsai

摘要

CRISPR-based genome editing therapeutics are entering the clinic, offering transformative potential but also presenting potential risks. Preclinical-to-clinical toolkits are needed to assess the safety and efficacy of these new therapies and accelerate progress. Emerging technologies to monitor the biological effects of genome editors cover a range of biological scales, from the direct measurement of editing outcomes in DNA, to human microphysiological systems, and non-invasive in vivo imaging. Measurements of on-target and off-target editing outcomes, including sequences unique to humans, provide essential benchmarks to understand functional responses. Microphysiological systems, including organoids and organs-on-chips, enable phenotypic evaluations of editing strategies in varied organ lineages and disease states. Non-invasive imaging modalities can track the biodistribution and activities of genome editors and edited cells in vivo. Collectively, these technologies provide complementary insights across different scales, from the single nucleotide to the whole organism, bridging preclinical therapeutics development with clinical trials.