SEVERE COMBINED IMMUNODEFICIENCY: NEW PERSPECTIVES IN GENE THERAPY

Abstract

Severe Combined Immunodeficiency (SCID) is a group of primary immunodeficiencies characterized by profound dysfunction in cellular and humoral immunity, resulting from mutations that compromise the development and functionality of T, B, and NK lymphocytes. Traditionally treated through hematopoietic stem cell transplantation, SCID has benefited significantly from recent advances in gene therapy, which offer safer and more effective alternatives for etiological correction. This study presents a narrative synthesis of the main technological innovations, clinical advances, and persistent challenges associated with gene therapy approaches, including the use of self-inactivating lentiviral vectors and gene editing techniques such as CRISPR/Cas9. The results demonstrate substantial improvements in immune reconstitution, a reduction in complications associated with conventional treatment, and a greater potential for definitive cure. However, issues related to genomic safety, cost, scalability, and equity of access still require attention. It is concluded that gene therapy represents a transformative and continuously evolving strategy for the management of SCID, pointing towards a new era of personalized and effective interventions.