Sunday, June 17, 2012

Gene Therapy Seems To Be Right Around The Corner

I had my first inkling of gene therapy way back when I saw Blade Runner in the theater (yes I'm that old). Warning: just play the first part of the clip if people having their eyeballs popped out offends you:



And yet despite the fact that artificial humans (how artificial are they when the have their own DNA-oh wait that's sort of the point) couldn't be saved by the genius Tyrell with gene therapy, in the real world scientists have already started implementing gene therapy to some extent. This is pretty amazing stuff. This is flying cars and fusion energy and lunar colonies scale cool.

Researchers have actually used retroviruses to alter DNA and cure diseases in the eye which is much more immune tolerant than the rest of the body. Awesome. Why am I just finding out about this now?
In a recent, 3-year follow up on 15 patients with Leber congenital amaurosis (LCA), a degenerative retinal disease that causes childhood blindness, Hauswirth and his colleagues found that within the area of the retina they are targeting for treatment, “patients have gained light sensitivity from as little as to 200-fold to as much as 60,000-fold,” he says. Twelve of the patients have also demonstrated significant improvement in visual acuity, reading an extra three lines lower on an eye chart, and in 13 of the patients, their pupils constricted when exposed to light as much as 100-fold dimmer, a more objective measure of light sensitivity.
This is still in the realm of sci-fi, but just barely. And it reminds me of an interesting story I recently read in Analog. A woman is unable to have children, she gets gene therapy to fix the problem but the resultant therapy has a knock-on effect and she has lost the desire to have kids and ends up divorcing her husband. The story was rather wooden but the very real fact of the extremely complicated nature of genetics, epigenetics and such a mind-bogglingly complicated system as the human body rang true.

1 comment:

  1. Exactly. This is why one should not rush to codify things into state control. You may end up with terrifying unintended consequences.

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