Regenerating an Immune System: Gene Therapy and Stem Cell Transplantation for Severe Combined Immune Deficiency

Abstract

Primary immunodeficiencies (PIDs) are a subset of immunologically based, inherited diseases that predispose affected individuals to a variety of infections, allergies, and autoimmune disorders, and leave them at risk of developing cancer. PIDs provide an invaluable model to dissect intrinsic mechanisms of the human immune system. Examination of these mostly monogenic conditions has lead to the identification of many of the associated genes and has produced a wealth of information about the development and function of the immune system. These pathologies give rise to immunological insights on the regulatory and genetic complexities of the innate and adaptive immune system and allow for the accreditation of scientific observations to underlying genotypes. Critical analysis and understanding of these disorders is essential as it fosters the development of new diagnostic and therapeutic tools for improved treatment of a wide variety of pathologies.