CRISPR-CAS9 IN THE TREATMENT OF INHERITED DISEASES: FROM LABORATORY TOOL TO CLINICAL PRACTICE

Over the past decade, CRISPR-Cas9 genome editing has undergone a remarkable transformation — from a foundational research tool to a tangible therapeutic strategy. This article reviews the core mechanisms of the CRISPR-Cas9 system and its derivatives — including base editing and prime editing — and examines clinical outcomes in the treatment of hereditary hemoglobinopathies, hereditary transthyretin amyloidosis, hereditary angioedema, and genetically driven cardiovascular disease. Particular attention is given to exagamglogene autotemcel (Casgevy), the first FDA-approved CRISPR-based therapy, alongside pressing challenges such as off-target editing, immune responses, and the delivery of therapeutic components to target tissues.