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'Bubble Boy' Cured by Gene Therapy in UK

Posted by timothy on from the heartening-and-lungening dept.

DrKyle writes "Another child with "Bubble Boy" disease aka ADA-SCID (adenosine deaminase deficiency causing severe combined immunodeficiency) has been cured by transforming bone marrow stem cells with the functioning gene. Normally toxic levels of adenosine build up in T-cells killing off those important cells required for a robust immune system. While not the first person cured, another successful case of gene therapy goes a long way in encouraging goverments to continue to fund genetic research."

4 of 56 comments (clear)

  1. Re:Important question by Doc+Ruby · · Score: 2, Insightful

    Wasn't your mother "playing god" by allowint your monkey body to live, and possibly breed, who genetically shouldn't make it past infancy, unable to feed yourself? Self-sufficient monkey or death - going halfway is stupid.

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    make install -not war

  2. Re:Playing God by thebatlab · · Score: 2, Insightful

    Not sure if you were being funny or real but...why is it assumed that it's god's will to have people lead these short tortured live's but not his will to give us the ability to cure them?

  3. Re:Important question by Jackazz · · Score: 2, Insightful
    What he means to say is, NO. In this case the bone marrow was removed, altered, and re-injected into the patient. The gene alteration was not systemic, did not effect the boy's sperm progenitor cells, and will not get passed on to his offspring.

    I'm not sure, but I bet this is a very rare gene and a recessive trait and if he mates with a normal individual, he will probably have normal children anyway.

  4. Re:Important question by John+Newman · · Score: 2, Insightful
    So, does the saved kid pass on the original deficiency in the gene pool?
    He passes on the gene, but not the disease. This form of SCID is a recessive disorder - you need two bad copies of the gene to get this disease. The alleles that cause disease are quite rare, and this kid won the reverse-lottery by having two parents who happened to both be carriers *and* both gave him their bad copy. He will certainly pass the disease allele onto his children, but his children would only have the disease if their mother was a carrier *and* gave them her bad copy. The odds of that are vanishingly low, and can be reduced to zero by genetically screening the mother (and if she is a carrier, doing IVF and screening the embryos).

    So there is little or no risk to his kids, and the "harm" done to the gene pool is minimal because the bad alleles are so rare anyway.