SCID-X1 - Healing Genes


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Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Phase 1 / 2


Researchers at Mattel Children’s Hospital of UCLA and Boston Children’s Hospital seek patients up to 5 years of age with SCID-X1 ) caused by mutations in the IL2RG gene. Researchers will withdraw stem cells, gene-modify them to deliver a normal copy of the IL2RG gene, and return them to the patient after a low dose of chemotherapy. It is hoped this will provide the patients with a normal immune system.

Study participants will undergo a blood draw treatment that sorts out specific cells and returns the blood to the patient. After the gene modification in the lab, the cells are infused to the patient post chemotherapy and the patient will be followed for 2 years after infusion of the drug. Required long-term monitoring for a total of 15 years after infusion will be performed on a separate protocol.


  • Be 2 months of age or older
  • Have a diagnosis of SCID-X1 based on immunophenotype and lack of T cell function
  • Meet lab assessments for minimal levels of the type of immune cells targeted by the gene therapy
  • Not have encephalopathy


  1. Screening before the treatment.
  2. Leukapheresis will draw the needed immune cells but return blood cells to the body.
  3. Researchers will use a virus in the lab to deliver the gene to the immune cells.
  4. A low dose of chemotherapy will be delivered.
  5. The gene-modified immune cells will be re-infused to the patient.
  6. The patient will be closely followed for 2 years, with less intensive follow up for 15 total years from beginning of study.


Trials will take place at the Mattel Children’s Hospital – UCLA, Map, and Boston Children’s Hospital, Map.

Contact: Colleen Dansereau  |  6179197008  |  [email protected]



David Williams

University of California, Los Angeles


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