SCID-X1 gene therapy

SCID-X1 gene therapy is an **autologous ex vivo hematopoietic stem cell gene therapy** for **X-linked severe combined immunodeficiency** caused by pathogenic variants in **IL2RG**, which encodes the common gamma chain used by multiple interleukin receptors. The approach typically involves harvesting a patient's **CD34-positive bone marrow stem cells**, transducing them outside the body with a viral vector carrying a functional **IL2RG** cassette, and reinfusing the corrected cells, often after **low-dose busulfan conditioning**, to reconstitute T-cell, B-cell, and natural killer cell immunity. Historically, early **gammaretroviral** versions demonstrated proof of concept but were associated with insertional oncogenesis, whereas newer **self-inactivating lentiviral vector** programs, including constructs such as **CL20-i4-EF1α-hγc-OPT**, were developed by academic groups including **St. Jude Children's Research Hospital**, **UCSF Benioff Children's Hospital**, and **Boston Children's Hospital** to improve safety while preserving durable immune reconstitution in infants lacking matched sibling donors.

Other names

anti-IL2RG autologous CD34-positive hematopoietic stem cellsanti-IL-2RG autologous CD34-positive hematopoietic stem cellsanti-IL 2RG autologous CD34-positive hematopoietic stem cellsautologous CD34+ hematopoietic stem cell gene therapy for SCID-X1IL2RG gene-corrected autologous hematopoietic stem cell therapyIL-2RG gene-corrected autologous hematopoietic stem cell therapyIL 2RG gene-corrected autologous hematopoietic stem cell therapylentiviral SCID-X1 gene therapy