Victor R. Andrade-Carmona, Torre de Consultorios, Hospital Angeles Lindavista, Mexico City, Mexico
María F. Arteaga-Álvarez, Servicio de Anestesiología, Hospital Regional 1º de Octubre, Instituto de Seguridad y Servicios Sociales de los Trabajadores del Estado, Mexico City, Mexico
CRISPR/Cas9 has moved from a gene-editing platform used in experimental biology to a clinically relevant therapeutic technology with approved and investigational applications in hematologic, hepatic, ophthalmologic, and cardiometabolic diseases. The objective of this brief review is to provide a clinically oriented synthesis of current CRISPR/Cas9-based therapies, summarize key efficacy and safety considerations, and discuss the translational requirements for implementation in Mexico and Latin America. A focused literature review was conducted using clinical-trial registries and peer-reviewed reviews of clinical translation, with emphasis on therapies that have reached human studies, approved products, pre-treatment testing, follow-up needs, and the Mexican institutional context. The available evidence shows that ex vivo editing for hemoglobinopathies has already reached regulatory approval, while in vivo editing programs such as NTLA-2001 and ocular CRISPR therapies remain in active clinical development. However, broad adoption in routine practice remains constrained by off-target risk assessment, immune and long-term safety monitoring, manufacturing complexity, and the need for multidisciplinary oversight. In Mexico, recent developments at UNAM, the Instituto Nacional de Pediatría, and IPN suggest growing technical capacity, but clinical translation will require coordinated regulatory, bioethical, laboratory, and referral frameworks. CRISPR/Cas9 should therefore be understood not as a universal intervention, but as a disease-specific therapeutic platform whose safe implementation depends on genotype confirmation, pre-treatment testing, structured follow-up, and institutional readiness.
Keywords: CRISPR/Cas9. Gene editing. Clinical translation. Mexico. Genomic medicine. Cell and gene therapy.
