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Gene Therapy for X-linked Chronic Granulomatous Disease
X-linked chronic granulomatous disease (X-CGD) is a rare genetic disorder, which affects boys. It is a primary immunodeficiency disorder which results from an inability of the white blood cells called phagocytic cells (or phagocytes) to kill invading bacteria and fungi. These cells have difficulty forming the free radicals (most importantly the superoxide radical due to defective phagocyte NADPH oxidase complex) which are important in the killing of ingested pathogens. In X-CGD (which accounts for two thirds of CGD patients), the defect lies in a gene which makes up a critical part of the NADPH-oxidase complex (the catalytic subunit; gp91-phox protein). Therefore they kill bacteria and fungi poorly, and the patients suffer from severe and recurrent infections. This also results in inflammation which can damage parts of the body such as the lung and gut. In many cases, patients can be adequately protected from infection by constant intake of antibiotics. However, in others, severe life-threatening infections break through. In some cases, inflammation in the bowel or urinary systems results in blockages which cannot be treated with antibiotics, and which may require the use of other drugs such as steroids. Development of curative treatments for CGD is therefore of great importance.
Details
| Lead sponsor | Genethon |
|---|---|
| Phase | PHASE1, PHASE2 |
| Status | TERMINATED |
| Enrolment | 3 |
| Start date | Mon Jun 24 2013 00:00:00 GMT+0000 (Coordinated Universal Time) |
| Completion | Fri Oct 17 2025 00:00:00 GMT+0000 (Coordinated Universal Time) |
Conditions
- X-Linked Chronic Granulomatous Disease
Interventions
- X vivo gene therapy
Countries
France