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

    Asked by icedancer to Gioia, Iain, Jo, Leo, Mariam on 25 Jun 2010 in Categories: . This question was also asked by fayeandcoral.
    • Photo: Gioia Cherubini

      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

    • Photo: Joanna Watson

      Joanna Watson answered on 18 Jun 2010:

      As the genes are the instructions for the whole body, in the future, gene therapy could probably be used for lots of things – like making people immune to certain diseases perhaps?

      I don’t know many gene therapies that are currently being used or tested, but there are some gene therapy treatments for Parkinson’s disease that are currently being tested to see whether they work and there is a trial of gene therapy for cystic fibrosis too. The other scientists might know of more.

    • Photo: Mariam Orme

      Mariam Orme answered on 22 Jun 2010:

      In theory, gene therapy could be used to treat any disease that occurs because of genes that are faulty. This would include pretty much any disease that isn’t caused by bacteria or viruses or some other pathogen. So the potential uses for gene therapy are huge.

    • Photo: Iain Moal

      Iain Moal answered on 24 Jun 2010:

      Gene therapy has been used on many different genetic illnesses, including cystic fibrosis, sickle-cell anemia, Parkinsons disease, Huntingtons disease and some blindnesses.

    • Photo: Leo Garcia

      Leo Garcia answered on 25 Jun 2010:

      Gene therapy is hoped to be used to treat diseases caused by problems in the genes, like cystic fibrosis, haemophilia and muscular dystrophy. For all of these, those, it is still early days for gene therapy, as it such a complex and difficult process. However, people are doing research into it every day, and progress is being made: