TREATMENT OF X-LINKED SEVERE COMBINED IMMUNODEFICIENCY VIA EX VIVO GENE THERAPY USING A LENTIVIRAL VECTOR COMBINED WITH NON-MYELOABLATIVE CONDITIONING

Abstract

In the light that lentiviral vectors have been deemed more efficient and safer at transducing hematopoietic stem cells making them the most promising option for SCID gene therapy, the project will explore a potential gene therapy treatment for X-linked SCID using a lentiviral vector.