what is gene therapy and when is it used?

Answer 1

For now, gene therapy means putting a good gene into someone who has a problem because of a bad one. For example, there are some people with SCID who essentially have no immune system (the bubble boy was one).

Researchers in France added a working gene to some babies' blood stem cells (in the bone marrow). These kids regained their immune systems and can now fight off disease. Unfortunately, 3 out of the 12 kids also got leukemia. Messing with genes is risky!

The genes are usually put in using a virus that has been made "safe" by getting rid of the harmful genes (the leukemia happened because the virus landed in a place that turned on a gene that causes cancer). Another way is to use naked DNA.

Right now scientists can't really go in and fix a gene. There are some techniques but none are really ready for primetime yet (see http://www.thetech.org/genetics/ask.php?id=49for the details).

Answer 2

Generally, using a retrovirus to replace portions of DNA that carry genetic disorders. There is some good information on the link below. Though there's a success story here, gene therapy as a checkered history, and comes with a lot of possible complications. For that reason, it is very rare, and currently only used when there are really no other treatments.

Answer 3

Gene therapy doesn't work on disease like diabetes. ONLY the "tendency" to get diabetes is genetically related, but diabetes istself is an AUTO-IMMUNE disease, unaffected by genetics. There is some current research using STEM CELLS as a potential CURE for diabetes. In this case, stem cells have been shown to CURE diabetes -- in lab mice! it is a LONG WAY between mice and men! Currently, this research is in the "verification" phase. this means that OTHER labs are trying to duplicate the results of the first lab. IF they are successful, then there is hope. The next step is to investigate the possibilities using human tissues -- like pancreases "donated to science". THEN, if the result are promising, they would begin testing on living human subjects. This last stage is called "Clinical Trials". afer the clinical trials, methods must be developed to harvest and "sort" the stem cells. Right now, this is VERY painful -- it requires drilling into your hip or leg bones to get the bone marow -- the best source for human stem cells. Finally, after YEARS of research, the "cure" needs FDA approval. For something this critical, you can expect a LOT of debates and question -- like 5-7 YEARS worth. A Stem Cells cure for diabetes -- if indeed there is a cure in this method -- is still 20-30 YEARS awasy. Note, too, that Stem Cell research is NOT gene therapy! The two things are COMPLETELY different! And of course, there is the potential moral and ethical problems surrounding Stem Cell Ressarch, but in the case of Diabtes Research it is easy. Fetal Stem Cells are NOT NEEDED! Adult Stem Cells work just fine, and are "harvested" from the patients themselves.

Answer 4

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what is gene therapy and when is it used?