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.
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.