Artemis-deficient-SCID - Healing Genes


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Autologous Gene Therapy for Artemis-Deficient SCID

A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells

Phase 1/2


Doctors seek patients 2 months of age and older with artemis-deficient Severe Combined Immunodeficiency (ART-SCID) caused by mutations in the DCLRE1C gene. Researchers will withdraw stem cells, gene-modify them to deliver a normal copy of the DCLRE1C 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 post chemotherapy and the patient will be closely assessed for 1 month, will follow up required at 42 days, 1 year, 2 years, and up to 15 years.


  • Be 2 months of age or older
  • Have a diagnosis of typical or leaky ART-SCID
  • Meet lab assessments for minimal levels of the type of immune cells targeted by the gene therapy
  • Not be pregnant or HIV-positive


  1. Screening before the treatment
  2. Blood apheresis using the CliniMACS system 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 assessed for 1 month, will follow up required at 42 days, 1 year, 2 years, and up to 15 years.


Trials will take place at the University of California, San Francisco Children’s Hospital. Map.

Contact: Morton Cowan, MD  |  [email protected]  | 415-476-2188

Contact: Jennifer Puck, MD  |  [email protected]  | 415 502-2090



University of California, San Francisco


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