Therapeutic uses of CRISPR-Cas9 in human medicine: an exploratory systematic review of clinical studies
Keywords:
CRISPR-Cas9; gene editing; human medicine; gene therapy; clinical trials; systematic reviewAbstract
Introduction: The CRISPR-Cas9 system has emerged as a revolutionary tool in molecular medicine, with growing interest in its therapeutic potential in humans.
Objetive: To describe the current use of the CRISPR-Cas9 system for diagnostic and therapeutic purposes in medicine.
Method: A systematic search was conducted in PubMed, Scopus, and Web of Science, with a cutoff date of July 5, 2025. Original clinical studies and indexed scientific reviews documenting the use of CRISPR-Cas9 in humans were included. The PRISMA 2020 criteria were applied, and studies not related to human interventions were excluded.
Results: The main conditions treated were hematological diseases, cancer, metabolic disorders, and hereditary diseases. Clinical benefits reported included transfusion independence, reduced crises, acceptable safety, and functional improvements, although challenges persisted regarding efficacy, gene delivery, and adverse effects.
Conclusions: CRISPR-Cas9 represents a significant step toward clinical application, but its widespread implementation still requires robust evidence, long-term follow-up, and regulatory frameworks that ensure safety and equity in medical use.
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