Chronic granulomatous disease - Healing Genes

Chronic granulomatous disease

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Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

A Phase I/II, Non Randomized, Multicenter, Open-Label Study of G1XCGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease

Phase 1 / 2


Doctors at research centers in CA, MD, and MA seek patients with X-linked Chronic Granulomatous Disease caused by mutations in the CYBB gene, to trial a gene-modified stem cell therapy designed to deliver a normal copy of the CYBB gene. Study participants will undergo a blood draw treatment that sorts out specific stem cells and returns the blood to the patient. After the gene modification in lab, the stem cells are re-infused post chemotherapy and the patient will be closely assessed during treatment, with follow-up for 2 years.


  • Be 23 months of age or older
  • Have a diagnosis of SCID-X1 confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 95% of the biochemical activity of the NADPH-oxidase
  • Not have a 10/10 HLA-matched donor available after searching of NMDP registries


  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 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 during treatment, with follow-up for 2 years.


Trials will take place at three research centers:

University of California, Los Angeles (UCLA) in Los Angeles, CA. Map.

The National Institutes of Health in Bethesda, MD. Map.

Boston Children’s Hospital in Boston, MA. Map.



Caroline Y Kuo, MD  |  310-794-1940  |  [email protected]

Kit L Shaw, MD  |  310-267-0584  |  [email protected]




University of California, Los Angeles

Boston Children’s Hospital

The National Institute of Allergy and Infectious Diseases (NIAID)


California Institute for Regenerative Medicine (CIRM)


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