Using gene therapy to treat chronic granulomatous disease (X-CGD)
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American and British teams led by Dr. Kohn (University of California, Los Angeles), Malech (NIH), Williams (Boston Children’s Hospital) and Trasher (Ormond Street Children’s Health Institute) in clinical medicine have shown definitive results of gene therapy for people with published chronic granulomatosis.
People with chronic granulomatosis or CGD have mutations in five genes that are involved in increasing the release of chemicals by white blood cells and fighting bacteria and fungi. Without this burst of chemicals, patients with this disease are prone to bacterial and fungal infections. The infection can be severe and life-threatening.
According to news-medical.net, the most common form of CGD is the so-called X-CGD, which affects only males and is caused by a mutation in the gene on the X chromosome. But in a new study, researchers used the potential of gene therapy and stem cells for nine patients with this condition, and six of them recovered and did not need to use other treatments.
Currently, besides the use of antibody courses, the only treatment option for patients with CGD is the use of a bone marrow transplant from a healthy and compatible donor, and the lack of suitable donors makes the treatment of this disease difficult. But in this new approach, researchers took hematopoietic stem cells from X-CGD patients and subjected them to genetic engineering in laboratory conditions and corrected their mutations. In the continuation of these genetic engineering stem cells that were able to produce appropriate white blood cells were transplanted into the body again and as it was said, 6 patients out of 9 patients who had problems have obtained almost complete recovery in this way. The way that the 3-year follow-up on them has indicated their complete treatment.
Access link to the article:
https://www.news-medical.net/news/20200128/Successful-gene-therapy-trial-in-patients-with-X-linked-Chronic-Septic-Granulomatosis.aspx
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This post is written by Saeid_Mohebbi