2006 National DNA Day Online Chatroom Transcript

This is just one question from an archive of the National DNA Day Moderated Chat held in April 2006. 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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Right now there are breakthroughs in organ transplants like the recent face transplants. Is it possible to create DNA transplants for those people with genetic diseases?
     Chris Austin, M.D.: Theoretically, yes, and this is known as "gene therapy". The idea is simple: deliver the correct gene (DNA) sequence to people who have a gene that is mutated. This has been going on for nearly 20 years, and is turning out to be much more difficult than anticipated. The reasons for this are, among other things, trouble getting the DNA sequences to enough cells in the right tissues (think of how difficult it is to get the right gene to all 10,000,000,000,000 cells in the brain!), and rejection from the immune system, that tends to see the correct gene (or protein for which it codes), or the "vector" that delivers the gene to the tissues, as foreign, and rejects it. The successes have been in blood disorders (cells are easy to reach) for disorders where only a few cells corrected is enough to help the person. An example of this is Severe Combined Immunodeficiency Syndrome, or SCID.
Haena Cho, Wootton High School


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