Personalized Immunotherapy for Congenital Immune Defects: Precision Approaches to Immune Reconstitution

Kagambira Zimbuga M.

Faculty of Medicine Kampala International University Uganda

ABSTRACT

Congenital immune defects, or primary immunodeficiency disorders (PIDs), represent a diverse group of genetically determined conditions that compromise immune function from birth. Recent advances in genomics, gene editing, and cell-based therapies have catalyzed a shift toward personalized immunotherapy—strategies that target the molecular root cause of each disorder while minimizing off-target effects. This review highlights the current landscape of precision approaches for immune reconstitution, including gene therapy, hematopoietic stem cell transplantation (HSCT), and targeted biologics. Gene therapy using lentiviral vectors and CRISPR/Cas9 has shown success in correcting defects in conditions such as SCID and Wiskott-Aldrich Syndrome. Innovations in HSCT, such as reduced-intensity conditioning and gene-modified autologous transplants, have enhanced safety and efficacy. Additionally, cellular therapies like adoptive T cells and regulatory T-cell infusions are gaining traction for immune modulation. Precision diagnostics—powered by next-generation sequencing and immune profiling—now guide therapy selection and monitoring. While challenges remain in access, long-term safety, and ethical oversight, personalized immunotherapy offers transformative potential for treating congenital immune defects. As the field progresses, integrating individualized interventions with global health equity will be key to maximizing impact and improving outcomes for affected children.

Keywords: Congenital immune defects, Personalized immunotherapy, Gene therapy, Hematopoietic stem cell transplantation, Immune

CITE AS: Kagambira Zimbuga M. (2025). Personalized Immunotherapy for Congenital Immune Defects: Precision Approaches to Immune Reconstitution. Research Output Journal of Engineering and Scientific Research 4(3): 95-101. https://doi.org/10.59298/ROJESR/2025/4.3.95101