Question: What else can Gene therapy be use for (other than cancer)?

Gioia Cherubini answered on 17 Jun 2010:

Hi icedancer,

to be honest, Gene Therapy wasn’t initially thought for cancer. The idea of Gene Therapy was that since at the basis of genetic diseases (for example cystic fibrosis, haemophilia and Duchenne muscular dystrophy) there is the mutation in one gene, the disease could be in principle cured by giving the normal form of the gene. The best vectors to deliver the “curative” gene inside the cells are viruses, because they evolved in million of years to be more efficient at inserting their DNA into the infected cell. A big problem of Gene Therapy for monogenetic diseases is that the gene is mutated in ALL the cells of your body, making it hard to deliver the right gene to ALL the cells for ALL your lifetime.
Later on, the realisation that also cancer is a genetic disease, even if more mutated genes are involved, made researchers think that Gene Therapy could be used also for the treatment of cancer. Infact, in this case only tumours cells have the mutated genes and in addition, the ultimate goal is to kill them, so you would need the expression of the right genes only for a limited amount of time in limited amount of cells.
Ops, this answer is a bit lengthier than I wanted!
If you are interested in Gene Therapy, here it is a useful link: http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml