Significant progress achieved over the past decade has turned gene therapy into a tangible and effective approach for treating and curing primary immunodeficiencies (PIDs).
What is Gene Therapy?
Gene therapy, akin to a ‘mini gene transplant,’ involves extracting stem cells from patients with primary immunodeficiency (PID), replacing the defective genes within these cells with healthy and fully functional genes, and reintroducing the gene-corrected cells back into the patient’s body. This process enables the cells to generate the necessary cells or proteins essential for …