Can gene therapy cure all genetic disorders (e.g. Cystic Fibrosis) or can it only cure some genetic disorders?

Question

Help appreciated.

Answer 1

More specifically, right now it doesn't cure anything at all: just about all gene therapy trials were put on hold years ago, because the dominant methodology (virii) sometimes worked, but often caused leukemias and other cancers, because the viruses integrated into the wrong place with alarming frequency.

New generation virii are well under way, and ought to be safer, because they will only integrate into specific sites in the host DNA.

When it does work, the easiest thing to cure will be recessive mutations, as the other posters have indicated. However, it is possible that with clever engineering, you could cure even dominantly acting mutations by, for instance, using inhibitors at lower points in the molecular pathways.

Answer 2

Theoretically, it could cure any recessive genetic disorder that is dependent on mutant alleles such as cystic fibrosis, sickle cell anemia, or muscular dystrophy.

It would not be able to treat structural disorders such as fragile X syndrome.

The current leading method, just inserting healthy copies, could not treat the handful of dominant genetic disorders such as Huntington's disease, but a method which trades the sick version for the healthy one would be able to cure these them.

Answer 3

Gene therapy has been a huge disappointment. It is a far more complex process than first conceptualized.

Answer 4

I think it mostlikely depends on the disorder.

Answer 5

ya i agree with kitty, depends on the disorder.

Answer 6

check out this research group that is trying to find cures for all kinds of disease they have a message board you can use to ask questions.............http://boinc.bakerlab.org/rosetta/