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April 24, 2026Molecular TherapyOpen Access

In vivo systematic detection of the outcomes of CRISPR/Cas9 mediated DNA repair in skeletal muscle stem cells

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Authors

LHLiangqiang HeYFYang FuZWZiliu Wang

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Overview

Systematic profiling of CRISPR/Cas9 outcomes in skeletal muscle stem cells reveals mechanisms of DNA repair, indicating implications for gene therapy safety.

Key Points

  • The research aims to explore the in vivo outcomes of CRISPR/Cas9-mediated DNA repair in skeletal muscle stem cells.
  • Utilized a CRISPR/Cas9/AAV9-sgRNA system to profile editing outcomes at 95 loci in skeletal muscle stem cells.
  • Characterized various types of DNA repair outcomes, including editing indels and large modifications.
  • Disrupted NHEJ and MMEJ pathways to assess their roles in regulating large on-target modifications.
  • Identified small editing indels and a high occurrence of large deletions and large insertions.
  • Large insertions included both exogenous AAV vector integrations and endogenous genomic DNA fragments.
  • Endogenous large insertions preferentially originate from active genomic regions influenced by chromatin architecture.

Cite This Study

He et al. (2026) studied this question.

synapsesocial.com/papers/69eb07a4553a5433e34b31adhttps://doi.org/10.1016/j.ymthe.2026.04.032
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1<i>In vivo</i> systematic detection of the outcomes of CRISPR/Cas9 mediated DNA repair in skeletal muscle stem cells2025
  2. 2Single-cell and in vivo profiling reveal heterogeneous and organ-specific CRISPR-Cas9 off-target and translocation outcomes2026
  3. 3Long-term in vivo CRISPR/Cas9 gene editing in liver triggers host immunity and clonal mutations in DNA Repair Genes2026
  4. 4Indel pattern-guided repair mapping reveals genome-wide DNA repair networks in CRISPR/Cas9 editing2026
  5. 5CRISPR/Cas9-Based Genome Editing: Understanding Differences in DNA Repair Pathways, Profiles, and Outcomes2026