Science Forum

Science Forum

  1. Joined
    06 Mar '12
    Moves
    642
    12 Nov '20 16:246 edits
    https://phys.org/news/2020-11-safer-crispr-gene-therapy.html

    they talk about it being used to target cancer, which is interesting. But I see no reason why this couldn't also be used, and via using X-rays rather than light to activate the liposomes, to selectively target, and selectively so to reduce the risk of severe side effects, just one single particular organ of the body that is badly effected by a mutation which functionally only badly effects that particular organ.
    For example; If someone has a harmful mutation that only significantly effects liver function and not other organs, this therapy could be used to relatively selectively correct the gene defect in just the liver. Although the X-rays would inadvertently shine through much of the tissue in front and behind the liver and thus that tissue would also be effected, most other tissue of the body won't be so and thus that still would help to reduce the risk of severe side effects.
  2. Joined
    20 Oct '06
    Moves
    8642
    21 Nov '20 00:04
    @humy

    As a cancer therapy this is not going to work. The efficiency of transfer and editing would need to be 100% and the number of genetic lesions in a tumor requiring repair are too many for one vector. However, for treating genetic diseases in which only a fraction of the cells in a tissue actually need to be edited, we already have great successes with CRISPR therapies.