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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."
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.
Christians do not have a problem with science or medicine. We have a problem with killing babies and using their body parts. There are now ways to produce stem cells without killing babies and so stem cell research is no longer an issue. The only Christians that have a problem with at least some medical technology (such as blood transfusions) are the Jehovah's Witnesses. They are a small minority Christian denomination.
The practical application of this form of treatment will (as all three papers do) use systemic, viral delivery, meaning all cells will be affected. You can modulate the probability a little by selecting a virus with affinity for particular tissues, but there will still be germline modifications. If you're really worried about that, you make vasectomy a required co-treatment.
It's still a long way from useful. All three papers report similar results: 2-5% changes in the DNA pool; 40-60% changes in the mRNA pool; 20-30% reduction in functional deficits. In a mouse model with a homogeneous genetic defect and without severe pathology
The results are pretty comparable to targeted exon skipping treatments, which started around 15 years ago and have made small human trials without serious adverse results. It's exciting to see research starting to make progress on fixing diseases where the root cause has been known for 40+ years, but the science (nevermind the FDA) is still 10+ years away.