articleMolecular Therapy — Nucleic AcidsJul 17, 2025GOLD OA

Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges

NKNechama KalterCFCarla Fuster‐GarcíaASAlfredo SilvaVRVíctor Ronco-DíazSRStefano Roncelli

Bar-Ilan University · University of Freiburg · +9 more institutions

PubMed
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Abstract

Targeted nucleases, primarily CRISPR-Cas-based systems, have revolutionized genome editing by enabling precise modification of target genes or transcripts. Many pre-clinical and clinical studies leverage this technology to develop treatments for human diseases; however, substantial off-target genotoxicity concerns delay its clinical translation. Despite the development of a wide array of tools, assays, and technologies aimed at identifying and quantifying off-target effects, the absence of standardized guidelines leads to inconsistent practices across studies. This review highlights the key challenges and potential solutions in ensuring the safety of gene editing studies for therapeutic applications, focusing…

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45
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28.48
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100%
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213
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Authors

11

Topics & keywords

Topics
Keywords
  • CRISPR
  • Genome editing
  • Computational biology
  • Biology
  • Human genome
  • Genome
  • Computer science
  • Genetics
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