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Related Experiment Video

Updated: Jun 25, 2025

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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Recent Therapeutic Gene Editing Applications to Genetic Disorders.

Eric Deneault1

  • 1Regulatory Research Division, Centre for Oncology, Radiopharmaceuticals and Research, Biologic and Radiopharmaceutical Drugs Directorate, Health Products and Food Branch, Health Canada, Ottawa, ON K1A 0K9, Canada.

Current Issues in Molecular Biology
|May 24, 2024
PubMed
Summary

Therapeutic gene editing, particularly CRISPR-Cas9, base editing, and prime editing, offers precise genome modification for genetic disorders. Innovations in delivery systems and new CRISPR variants enhance efficacy and safety for precision medicine applications.

Keywords:
CRISPRgene editinggenetic disordersoff-target

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Area of Science:

  • Genetics and Genomics
  • Biotechnology
  • Molecular Biology

Background:

  • Therapeutic gene editing has advanced significantly, transforming the treatment of genetic disorders.
  • The clustered regularly interspaced short palindromic repeats (CRISPR) technology has emerged as a key tool for precise human genome modification.

Purpose of the Study:

  • To review the milestones and advancements in therapeutic gene editing technologies.
  • To highlight the impact of CRISPR-Cas9, base editing, and prime editing on treating genetic disorders.
  • To discuss the role of enhanced delivery systems and novel CRISPR variants in clinical applications.

Main Methods:

  • Comprehensive review of recent literature on therapeutic gene editing.
  • Analysis of CRISPR-Cas9, base editing, and prime editing technologies.
  • Evaluation of delivery systems (viral vectors, nanoparticles) and newer CRISPR variants (Cas12, Cas13).

Main Results:

  • CRISPR-Cas9, base editing, and prime editing demonstrate high precision and efficacy in ex vivo and in vivo genomic modifications.
  • Advanced delivery systems improve the safety and clinical translatability of gene editing therapies.
  • Novel CRISPR variants and prime editing offer enhanced versatility and reduced off-target effects.

Conclusions:

  • Therapeutic gene editing innovations are enabling treatments for a wide range of genetic disorders, from monogenic to polygenic conditions.
  • Breakthrough technologies are advancing preclinical and clinical trials, integrating into precision medicine.
  • Continued research is essential to address challenges like off-target effects and ethical considerations.