lightreads: a partial image of a etymology tree for the Indo-European word 'leuk done in white neon on black'; in the lower left is (Default)
lightreads ([personal profile] lightreads) wrote 2018-11-25 03:13 pm (UTC)

Yes. The gene therapy approved last year that is relevant to me has been in trial for....12 years? I knew about it, vaguely. I wasn't eligible for the trials because I'd had a traumatic retinal detachment, but I'd hear something about it every year or two and think oh yeah. They're still at that? But never actually expecting anything to come of it.

And then last winter there I was, minding my own business taking a bath and listening to an economics pocast. And in passing one of the hosts mentioned this brand new, first of its kind gene therapy for a rare eye condition. And I thought what are the odds? But yeah, there it was. Only applicable to a very specific mutation that 8% of the diagnosed population has, but. It's real and it works. Apparently they picked this one rare thing because it was, for some reason beyond me, a simplified method to work out how to do this, thus clearing the way for their bigger target, hemophilia. Which they have also cured, I believe.

But yeah. It's uncomfortable knowing we are on the brink of a huge explosion in this field and not knowing how to scope what that could be. And how long it will take. And what it's limitations will be. But it's definitely coming now, that's not science fiction anymore.


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