Gene Therapy

Hundreds of thousands of people with failing eyesight have been given fresh hope of a cure after gene therapy techniques were used to treat a teenager.

The treatment transformed the life of a severely visually impaired 18-year-old. Healthy genes were injected into one eye, leading to a significant improvement.

Eh? How does this work? I was under the (obviously mistaken) impression that gene therapy only worked if you could get it into the cells that then replicated. That is, that you needed to get it into the actual genetic structure of the individual and that thus such therapy really only worked upon hte next generation.

Clearly, I\’m wrong, but seriously? Just inject the healthy genes into the afflicted organ and everything starts to work?

5 thoughts on “Gene Therapy”

  1. This kind of treatment can provide a purely temporary “cure”. It is often used to give relief to cystic fibrosis sufferers: they inhale a spray of healthy lung cells which then coat the sufferer’s lungs and reduce the amount of mucus being secreted.

    However, it is a temporary measure only: once the cells die, then you need to repeat the treatment…


  2. David B. Wildgoose

    Gene Therapy that has its effect upon subsequent generations is known as “Germ-Line Therapy” (for obvious reasons).

    However, I was under the impression that this treatment shouldn’t necessarily need to be repeated because it is a correction to a faulty gene in the individual’s cells. When these cells create copies of themselves they should automatically also copy the inserted (corrected) gene. This is no different to the process whereby a copying error that results in cancer is also reproduced faithfully, including the mistake(s) that caused the cancer in the first place.

  3. The gene to be inserted is carried in a virus which infects the faulty cells – and thus gets into their “genetic structure”, in this case in the retina.

    There is no need to repeat the treatment since the “infected” cells containing the now healthy gene undergo mitosis in the body, just like any others and the gene continues to be present and do its job – in this case enabling the cells that provide night vision to work correctly.

    In this case it has worked spectacularly well, the lad was only given the lowest dose as a test, to ensure there were no side-effects such as unwanted immune responses. They were not actually expecting this result.

    It’ll still be at least 5-7 years, before it becomes anywhere near the commercially viable stage – manufacturing genes is still very expensive until we reach the next technological leap in protein synthesis.

    What a remarkable outcome for this young chap. Brightens one’s day immensely.

  4. It is a result to lift the spirits of the most jaded. It’s so good, I’m expecting some creep to try to ban it now.

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