2011 National DNA Day Online Chatroom Transcript

This is just one question from an archive of the National DNA Day Moderated Chat held in April 2011. The NHGRI Director and many genomics experts from across NHGRI took questions from students, teachers and the general public on topics ranging from basic genomic research, to the genetic basis of disease, to ethical questions about genetic privacy.

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What are some of the latest successes with gene therapy?
     Pam Schwartzberg, M.D., Ph.D.: I currently study mouse models of primary immunodeficiencies (genetic diseases that impair the ability of the immune system to fight infections) and how specific disorders affect responses to infectious diseases. There have been some spectacular successes with gene therapy, particularly for some of the genetic disease associated with vision, where vision has been restored in Leber congenital amaurosis. The eye is particularly great for delivery of genes. But there are also successes in the field of immunodeficiencies and other disease of the blood. And the use of gene therapy for metabolic disorders is a promising new area (in mouse models).
Lexington High School in SC (10th grade student)

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