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Medical Gene Therapy Cures Sickle Cell Disease* (*in one patient, for at least 15 months)

  1. Mar 3, 2017 #1


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    *in one patient, for at least 15 months

    Research published this week in the New England Journal of Medicine reports on a case of using gene therapy to potentially cure a teenage boy of sickle cell anemia, a genetic disease that impairs red blood cell function:

    Of course, there are limitations to the study. They treated only one patient and have only monitored the patient for 15 months. Additional follow up will be required to determine whether the genetically modified blood stem cells will persist in the patient's body over many years. Furthermore, additional testing on many more patients will be required to establish the safety and efficacy of the treatment. In particular, the method of gene modification, lentiviral transduction, will insert the corrected β-globin gene randomly into the blood stem cells. This random insertion carries the risk of disrupting important genes in the blood cells to cause diseases like cancer. Newer, more precise gene editing methods like CRISPR could minimize these risks in future trials.

    The results are a very nice proof of principle for gene therapy, especially after some very tragic failures in the past. However, despite the success in this initial trial, much more work needs to be done before this method of gene therapy will be ready to enter routine clinical use.

    Scientific publication: http://www.nejm.org/doi/full/10.1056/NEJMoa1609677#t=article
    Popular press summary: http://www.chicagotribune.com/lifes...y-sickle-cell-health-0308-20170303-story.html
  2. jcsd
  3. Mar 6, 2017 #2
    Why didn't they use CRISPR?
  4. Mar 6, 2017 #3


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    Not sure. This therapy probably had been in the works for many years before CRISPR was invented, so they probably just went forward with what they had been working on. Patent and licensing issues could have also been involved as the research was sponsored by a biotech company that hopes to commercialize the therapy.
  5. Mar 9, 2017 #4


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    I came across an interesting article on a group developing a machine to automate much of the gene therapy process in order to make it more accessible to people in the third world:
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