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by reasonattlm
3341 days ago
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It is worth noting that the real challenge in gene therapy at this time is not editing the genes, but in getting sufficient coverage of edited cells (and especially progenitors and stem cells) in an adult individual to achieve the therapeutic goal and make it last. (Most studies of genetic alterations in the broader sense have bypassed this challenge by working with animal lineages, or animals in which the editing happens in the earliest stages of development, when there are very few cells needing to be changed. The alteration then propagates during embryonic and later development). There have been a number of very promising studies in the past year or two with regard to gene therapies to apply to adults, such as animal studies that demonstrated a cure for an inherited muscular dystrophy, but in the bigger picture, comprehensive coverage of tissues and cells is still something that the research community is in the midst of getting to grips with. |
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The way I understand the problem is that the lag time can be rather long, and it's always long enough for the virus to get a good running start. Symptoms, after all, don't occur until things are well under way. So in the real world, the two opportunities for antiviral therapies are (1) something that you can take long before you're even exposed, and that lasts for a long time (like a vaccine) or (2) something that you can take after you've already realized that you're sick (like an antiviral drug).
It seems like the DRACO proteins fall in between these two unless something has changed since the last time I read about this in, I believe, 2012.