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Pop karnchanapimonkul

Sight Seen: Gene Therapy Restores Vision in Both Eyes: Scientific American - 0 views

www.scientificamerican.com/article.cfm

gene therapy vision disease enzyme

shared by Pop karnchanapimonkul on 13 Apr 12 - No Cached
  • gene therapy to treat blindness in 12 adults and children with Leber's congenital amaurosis (LCA), a rare inherited eye disease that destroys vision by killing photoreceptors—light-sensitive cells in the retina at the back of the eye.
  • genetic mutations in retinal cells. One mutated gene that causes the disorder is named RPE65. An enzyme encoded by RPE65 helps break down a derivative of vitamin A called retinol into a substance that photoreceptors need to detect light and send signals to the brain.
  • injected a harmless virus carrying normal copies of RPE65
  • ...8 more annotations...
  • subsequently began producing the enzyme
  • proved so much they no longer met the criteria for legal blindness
  • injected the functional genes into the previously untreated eye
  • improved as soon as two weeks after the operation: They could navigate an obstacle course, even in dim light, avoiding objects that had tripped them up before, as well as recognize people's faces and read large signs
  • brains were much more responsive to optical input as well.
  • second round of gene therapy further strengthened the brain's response to the initially treated eye as well as the newly treated one
  • that neuroplasticity plays a role
  • visual cortex responding to the newly flowing channel of information from the second eye bolster activity in areas of the visual cortex responding to the initially treated eye.
  • Pop karnchanapimonkul on 13 Apr 12
     
    Article about how the enzyme produced from gene therapy is used to cure blindness in an eye genetic disease.
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