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July 25, 2025Molecular Therapy — Nucleic AcidsOpen Access

Recent applications, future perspectives, and limitations of the CRISPR-Cas system

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Authors

SPSun-Ji ParkGLGyeongja J. LeeSCSoo Min Cho

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Overview

Review discusses CRISPR-Cas advances in gene therapy for genetic defects, highlighting challenges like off-target activity.

Key Points

  • CRISPR-Cas system allows precise genome editing, correcting pathogenic mutations effectively.
  • It introduces double-strand breaks to facilitate gene corrections through various repair mechanisms.
  • The review covers CRISPR's applications in modeling diseases and potential therapeutic uses for hereditary disorders.
  • Challenges such as off-target activity and delivery issues are addressed, with strategies proposed to enhance efficacy.

Cite This Study

Park et al. (2025) studied this question.

synapsesocial.com/papers/689a0933e6551bb0af8ce4c8https://doi.org/10.1016/j.omtn.2025.102634
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