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Biotech Medicine

Gene Editing Offers Hope For Treating Duchenne Muscular Dystrophy ( 48

schwit1 writes with news that scientists have used a new gene-editing technique called CRISPR to treat mice with defective dystrophin genes. This is the first time that such a method has successfully treated a genetic disease inside a living mammal. The Times reports: "Three research groups, working independently of one another, reported in the journal Science that they had used the Crispr-Cas9 technique to treat mice with a defective dystrophin gene. Each group loaded the DNA-cutting system onto a virus that infected the mice's muscle cells, and excised from the gene a defective stretch of DNA known as an exon. Without the defective exon, the muscle cells made a shortened dystrophin protein that was nonetheless functional, giving all of the mice more strength."
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Gene Editing Offers Hope For Treating Duchenne Muscular Dystrophy

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  • It will be only months until it will be used for some athlete's "Muscular Dystrophy", then we'll know if it's safe.

    • by Anonymous Coward

      Actually, no.
      It helps sick people by removing faulty part of the DNA. If you aren't sick, in best case, it'll make your scores worse.

    • The trouble with athelete-research is that it is nominally wicked and immoral, so everybody carefully avoids keeping useful records about the experiments.

      It's a real pity, there would probably be some very interesting stuff there if the dataset were available. At least the Purity of Sport remains unsullied, so there's that.
  • Quirks and Quarks (Score:3, Informative)

    by lazarus ( 2879 ) on Monday January 04, 2016 @08:36AM (#51234165) Homepage Journal

    Quirks and Quarks did a podcast [] very recently about this technology and its application on a particular strain of MD. This work was done (by Dr. Ronald Cohn from the Hospital for Sick Children in Toronto) on living cells however, not live mammals. The podcast does go into a high level and easily understood description of how the technology works. Fascinating stuff.

    • Radiolab also did a netcast [] on this.

    • I would have like to have up-voted the link to more information, both podcasts are interesting.

      There are many lectures on YouTube requiring varying degrees of expertise to understand but this one seems comprehensible to anyone with a STEM background and I recommend you take a look. Jennifer Doudna. The CRISPR-Cas 9 Genome Engineering Revolution, UC Berkeley Events channel (one of many excellent primary sources on YouTube) []

      I appreciate that many people with an interest will us

  • by jeffb (2.718) ( 1189693 ) on Monday January 04, 2016 @08:51AM (#51234213)

    I know there are concerns around human genetic manipulation, but there are a lot of people suffering in its absence. I'd be willing to take the risk on a therapy like this if I were suffering from a debilitating or fatal genetic illness. Furthermore, I am ready to shoulder my portion of the societal and ethical risks entailed by others testing it.

  • So would this also effect the male sex cells post gene therapy? Meaning, would the progeny still inherit the defective gene?

    • by Qzukk ( 229616 )

      It would depend on the ability of the virus to infect them. Since viruses tend to destroy the host cells when they replicate (which would make the gene splicing useless since the fixed cell is now dead), I would assume that this particular virus would be engineered to not replicate, preventing it from spreading through the body.

      • This is true - at the moment, there is no significant research using replicating gene therapy vectors for treating genetic diseases. Anti-cancer gene therapy vectors often are replication-competent, but for treating things like DMD or hemophilia the vectors cannot replicate.
    • At the moment, the FDA prohibits any gene therapy with a significant chance of germ line effects. So, for now, people with DMD who get gene therapy would still pass on their mutation. In principle, we could design something with intentional germ line effects, but that gets a lot trickier and we aren't sure enough about the safety for that yet.
      • by sjames ( 1099 )

        I'm not so sure the concern is even valid in this case given that the germline is already compromised.

    • The summary indicates that this virus infects muscle tissue. I don't know how selective a virus can be, so I don't know how effectively you could select one that specifically targeted or refrained from targeting germ cells or their progenitors.

      It seems that making a modification that can be carried down the female line would be trickier, since egg cells are all produced long before puberty begins. You'd need to infect those cells directly, I'd think, and I imagine any such therapy would lag well behind the

  • CRISPR is about to bring us an avalanche of genetic engineering on the human genome to attack a variety of gene-related diseases. This is our chance to eliminate the anti- science moment the way Darwin intended, by selecting out people who oppose GMO technology. Good riddance!

    • by jedidiah ( 1196 )

      Yes. Because releasing a bunch of little frankensteins out into the wild is the same as keeping them confined to a single human host. You are ever so intellectually superior for being willing to completely ignore these distinctions, or consider the trustworthiness of exactly WHO is using a particular technology. Your scientific hubris is simply fabulous.

      • When it comes to fighting something like genetic disease, we need more scientific hubris. We need more of the old sense of adventure that once made us first in applying science to real-world problems. If we want society to fund more research, public or private, we need to make more use of the results research provides.

The young lady had an unusual list, Linked in part to a structural weakness. She set no preconditions.