Scientists Cure Genetic Blindness

I, for one, feel like every time I turn on the television doctors are giving me bad news...

“A, B, C AND D all cause cancer.”

“You’re probably pre-disposed to high cholesterol.”

“I BET YOUR COLON IS UNHEALTHY!”

And all the so called medical breakthroughs seem to be for ridiculous things. I know you’re as relieved as I am that I can finally cure my dog’s restless leg syndrome! Don’t get me wrong, I’m not trying to discount all the amazing things that doctors and medical researchers do, I’m just happy to have finally read something that is going to make an actual difference in people lives, not just make them consult a doctor if they’ve had an erection lasting four hours or more (though rare, those require immediate medical attention).

Researchers have discovered a gene therapy treatment that actually cures some forms of genetic blindness. This therapy treatment increases cone and rod photoreceptor-based vision.

(Image Source)

Mutations is the RPE65 gene disrupt the retinoid cycle and cause a congenital human blindness known as Leber congenital amaurosis (LCA), an incurable retinal degenerative disease. The scientists used virus based gene replacement therapy to treat three young adults with RPE65-LCA and measured the results up to 90 days.

Within 30 days of the therapy, all three patients showed a statistically significant increase in visual sensitivity. Although the reconstructed retinoid cycle was not completely perfect, the new rods were very slow, the results were still dramatic; children who were once blind now could see.

(That last line was not meant to be pulled out of Amazing Grace lyrics. I apologize for that.)

(If you’re interested: clinical trial — abstract — paper — ABC News video).