2009 National DNA Day Online Chatroom Transcript

This is just one question from an archive of the National DNA Day Moderated Chat held in April 2009. 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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21

What are the problems that are currently preventing the effective use of gene therapy in humans?      David Bodine, M.D., Ph.D.: I investigate the genetics of pluripotent hematopoietic stem cells (PHSCs) to improve the effectiveness of bone marrow transplantation and to find better ways to use these unique cells for gene replacement therapy. Very few. Although the news papers have not been reporting the successes like they do the problems, the last two years have shown very successful applications of gene therapy. in Cancer Gene therapy has completely irradicated several different kinds of tumors and I predict that there will be FDA approved cancer gene therapy in a year Kids who were blind have had their sight restored by gene therapy and the immune deficient kids (even those you might have heard about that got sick) are all well now. The two biggest problems we have are that sometimes when you make a new protein in a patient, their immune system recognizes it as foreign, and the other is to make sure that we don't cause an unexpected disease while we are curing one. Saint Ignatius High School in IL (student)

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