CRISPR-Based Genome Editing in Contemporary Clinical Medicine: Therapeutic Translation, Global Challenges and the Future of Precision Medicine
TL;DR
CRISPR has progressed from an experimental genome-engineering technology to a clinically relevant therapeutic platform, although its future impact will depend on the ability to combine molecular precision and durable therapeutic benefit with rigorous safety assessment, responsible governance, and equitable access across diverse populations and healthcare systems.
Abstract
CRISPR-based genome editing has transformed the therapeutic possibilities of genomic medicine by enabling programmable modification of disease-associated genes and regulatory pathways. This review analyzed the evolution, current clinical applications, emerging therapeutic strategies, safety limitations, ethical challenges, and future perspectives of CRISPR-based technologies within an international framework that incorporates Latin America. An integrative literature review was conducted using scientific evidence published between 2012 and July 2026, with emphasis on peer-reviewed studies indexed in major biomedical databases and relevant international and regulatory documents. The evidence showed a rapid progression from foundational CRISPR-Cas9 research toward base editing, prime editing, ex vivo cellular modification, and direct in vivo genome editing. Clinical translation was most advanced in hematology, particularly in sickle cell disease and transfusion-dependent β-thalassemia, where genome-edited autologous hematopoietic stem and progenitor cells have demonstrated clinically meaningful therapeutic outcomes. Applications in oncology and hereditary transthyretin amyloidosis further demonstrated the feasibility of engineered immune cells and systemic in vivo genome editing. However, broader clinical implementation remains limited by delivery and tissue targeting, off-target and unintended on-target modifications, immunogenicity, manufacturing complexity, long-term safety, regulatory requirements, and accessibility. The international analysis identified heterogeneous capacity for clinical implementation, emphasizing the need to strengthen genomic infrastructure, population representation, professional training, regulatory preparedness, and research participation in Latin America. CRISPR has therefore progressed from an experimental genome-engineering technology to a clinically relevant therapeutic platform, although its future impact will depend on the ability to combine molecular precision and durable therapeutic benefit with rigorous safety assessment, responsible governance, and equitable access across diverse populations and healthcare systems.