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Gene Editing Offers Hope For Treating Duchenne Muscular Dystrophy (nytimes.com)
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."
There's a lot of "hope" for new technologies in the medical field. However, they'll be suppressed, banned, delayed, etc due to:
1) FDA endless bitching and bureaucratic process that requires 20-30 years of work to get approval.
2) Religious nutcases will lobby for a ban on this. Because God doesn't want you messing with genetics.... he only wants your donations.
It will be only months until it will be used for some athlete's "Muscular Dystrophy", then we'll know if it's safe.
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.
I am not interested in articles about life extension advancements.
2)Remember the religious furor over stem cell research? The same God didn't make it mantra faded quickly when the technology began to pan out. Turns out, a potential cure in the hand for a loved one wins out over some message interpreted from a thousand-year-old-tome.
Happiness in intelligent people is the rarest thing I know.
Ernest Hemingway
Then he said the hell with it all. Moved to France to live the heroes life. And then he died.
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?
Life is not for the lazy.
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!
We have a problem with killing babies and using their body parts.
So follow Planned Parenthood's example, and don't kill babies for body parts. Oh wait, you can't tell the difference between an acorn and a tree, or a fetus and a living baby, and you bought into that long-since debunked, fraudulent video that has incited so much anti-woman and anti-choice violence of late.
There are now ways to produce stem cells without killing babies
Yes, like from aborted fetuses, which as anyone with a scientific background can explain to you, is not the same thing as a baby.
You sir are not open to science, you are merely a religious zealot doing a poor job of trying to imitate one.
We were told about the CRISPR/CAS9 approach being tested in animals, but as you note, it is even further down the road then anti-sense therapies. Right now we have to hope that our son can get access to the anti-sense therapies in time and that this will buy him the time he needs for the CRISPER/CAS9 approach to get approved.
The part of it that is eating us up every day is that, even our most optimistic guess for when each therapy will become available isn't soon enough for even our most optimistic prognosis for his condition. The most likely outcome is that he will be in the wheelchair full time and require assistance eating before the anti-sense therapy becomes available and that he will be dead just before the CRISPR/CAS9 "cure" gets full approval.
I need a wheelchair van for my son. Help me get the word out. https://www.gofundme.com/wheelchair-van-for-jj
My son has Duchennes Muscular Dystrophy. He's 10 now and we've been waiting for the exon skipping anti-sense therapy trial you referred to for over a year now. Canadian stage 1 trials were just concluding when we got the diagnosis, we've been told he will only be accepted into the stage 2 trial (when it finally opens) if he is still walking. So now we are in the grim race to keep him ambulatory long enough to qualify for the trial whenever that happens to be. Even the researcher himself cannot give us even a loose estimate of how that will take, since stage 2 trials will only get approved after the stage 1 data has been crunched and reviewed and crunched again by Health Canada.
We were told about the CRISPR/CAS9 approach being tested in animals, but as you note, it is even further down the road then anti-sense therapies. Right now we have to hope that our son can get access to the anti-sense therapies in time and that this will buy him the time he needs for the CRISPER/CAS9 approach to get approved.
The part of it that is eating us up every day is that, even our most optimistic guess for when each therapy will become available isn't soon enough for even our most optimistic prognosis for his condition. The most likely outcome is that he will be in the wheelchair full time and require assistance eating before the anti-sense therapy becomes available and that he will be dead just before the CRISPR/CAS9 "cure" gets full approval.
I truly thank you for your comment and hope that, if not these avenues of treatment, that another one might become available in time. My husband, (with a similar deteriorating if not fatal form of muscular dystrophy), constantly studies medical papers all the time to find out the stages of research. We know the pain of hope for a treatment only to find it canceled due to market concerns, i.e. not enough profit can be made from the drug for the margin to be as popular as say diet pills. Duchennes is by far the most time critical form of muscular dystrophy that needs a cure now instead of later. I wonder how so many people can push the idea of a fatal genetic destiny predetermined by god when so many of their failings and accidents are treated only through the benefit of medical science. Let's be honest and say that ethics for treatments should be better left to the professionals who know what a treatment is, how it is put together, and all the actual potential side effects. Not the common conspiracy theorists who might be better spending their time saying they were abducted by aliens than that dead babies went into a treatment and vaccines gave them autism.