2008 National DNA Day Online Chatroom Transcript
This is just one question from an archive of the National DNA Day Moderated Chat held in April 2008. 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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How does gene therapy work?
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Donna Krasnewich, M.D., Ph.D.:
Investigating diseases resulting from abnormal metabolism of carbohydrates.
Gene therapy is used to treat genetic disorders by a very special medical strategy. Because we know that many disorders are caused by changes in individual genes, the strategy is to put a gene in the body that has normal sequence and function. There have been many attempts at this with the problem being: the gene must make enough of the normal protein to cure the disease, also, sometimes it is really difficult to get the gene to go to cells that have the disease. For example, gene therapy for a cystic fibrosis, that affects the lungs, must be directed toward lung cells. So far, the greatest successes in gene therapy have been in disorders of the immune system where the patient's own bone marrow cells, which contain immune cells, are withdrawn from the body, the functional gene is inserted, and the bone marrow is given back to the patient. Hopefully, these new cells, with the gene inserted, will function and help the patient feel better. |
| Scarlet Oaks in OH (12th grade student) |
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