Chronic Granulomatous Disease - Research

Research

Gene therapy is currently being studied as a possible treatment for chronic granulomatous disease. CGD is well-suited for gene therapy since it is caused by a mutation in single gene which only affects one body system (the hematopoietic system). Viruses have been used to deliver a normal gp91 gene to rats with a mutation in this gene, and subsequently the phagocytes in these rats were able to produce oxygen radicals.

In 2006, two human patients with X-linked chronic granulomatous disease underwent gene therapy and blood cell precursor stem cell transplantation to their bone marrow. Both patients recovered from their CGD, clearing pre-existing infections and demonstrating increased oxidase activity in their neutrophils. However, long-term complications and efficacy of this therapy are unknown.

Read more about this topic:  Chronic Granulomatous Disease

Famous quotes containing the word research:

    After all, the ultimate goal of all research is not objectivity, but truth.
    Helene Deutsch (1884–1982)

    If politics is the art of the possible, research is surely the art of the soluble. Both are immensely practical-minded affairs.
    Peter B. Medawar (1915–1987)

    One of the most important findings to come out of our research is that being where you want to be is good for you. We found a very strong correlation between preferring the role you are in and well-being. The homemaker who is at home because she likes that “job,” because it meets her own desires and needs, tends to feel good about her life. The woman at work who wants to be there also rates high in well-being.
    Grace Baruch (20th century)