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Saturday, November 22, 2008
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Primary Immunodefiency Diseases

Future Directions-Working Toward A Cure

Primary immunodeficiency diseases research will be focused on several goals in the coming years:

  • Develop the technology to make gene transfer an effective and curative therapy

  • Overcome transplant rejection to make bone marrow transplantation from HLA-matched nonfamily members reliable

  • Identify additional gene defects that cause primary immunodeficiency diseases

  • In primary immunodeficiency diseases not caused by a single gene defect, identify the immunologic abnormalities that lead to defective function


Gene Therapy
Patient with chronic granulomatous disease
participating in a clinical trial of gene therapy

  • Identification of new immune system molecules and their function allows development of new treatments for autoimmune diseases (diseases in which the immune system mistakenly attacks the body's own cells and tissues)

  • Improved understanding of how to regulate the immune system allows for the development of improved vaccines

  • Development of gene transfer technology will be useful in treating cancer and preventing rejection of transplanted organs and tissues

  • Development of new antibiotics will be useful for everyone in an era of drug-resistant germs


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