Community | October 25, 2009 | 7 comments

Study shows hope for type of blindness

source: http://online.wsj.com/article/SB125640173237005925.html

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tcmfan08
A small but provocative study showed that a form of gene therapy significantly improved the vision of patients left legally blind by a rare genetic eye disease. The benefit was especially striking among children.

Researchers said the findings amount to an important advance toward medicine's ambitious but generally unrealized dream of replacing disease-causing mutant or missing genes with normal DNA to treat and cure debilitating illnesses

In the study, 12 patients, including four children between ages 8 and 11 years old, underwent a surgical procedure in which a gene that makes a protein critical to vision function was injected into one eye. The second eye wasn't treated. While normal sight wasn't restored in any of the patients, all reported some improvement. Six gained enough vision that they may no longer meet criteria for being legally blind, researchers said, including the four children for whom substantial recovery of vision appears to have transformed their lives.

The children "were using Braille" in school before they were treated with the gene therapy, said Katherine A. High, co-lead investigator for the study. "Now they're reading books."

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  1. groups:
    Community,   Current Tonight,   Picked for Current Tonight,   Studies
  2. tags:
    Gene Therapy,  Blindess
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7 comments // Study shows hope for type of blindness

  • RFIDemocracy
    • 0
      RFIDemocracy  
    • Image

    • Very good development, should it prove fruitful in the long run.
      Also noteworthy is this recent article on 'neural stem cells'.

      The author notes: Currently, gene therapy has been leading the charge in new treatments for vision disorders. However, the conditions for which there has been the most progress, including Leber congenital amaurosis (LCA), have relatively few sufferers. Aside from a small population base of potential beneficiaries, gene therapy is largely assumed to be most effective if administered during the early stages of a disease. Looking into the future, Qiu notes, stem cell treatment could be a good alternative for those who might have already missed the window for effective gene therapy.

    • 2 years ago
  • Progresshiv
    • 0
      Progresshiv  

    • There are so many wonderful treatments that can be developed through genetic experimentation. I hope that this treatment will be used to help many more folks see well enough to read. The funding will be a key component of that.

    • 2 years ago
  • Gravity_Man
    • 0
      Gravity_Man  

    • Many blind people will fool sighted folks because being blind they're forced to over-develop the rest of their brain. It initiates a v/significant IQ jump. The blind have been used by the military for their ability to recall detailed facts from conversations. They're a cut above normal unless of course they were born with other issues. That sometimes happens. I have two cousins, both born with retinitis pigmentosa, so I don't imagine this would help them. Their optic nerve was atrophied. They said the oldest was retarded but he was playing possum. Pete didn't want to leave home every year to attend school.

    • 2 years ago
  • larrysnotes
  • J_Jammer
  • Gravity_Man
    • 0
      Gravity_Man  

    • I was hopeful til I came to "no adverse side effects". Bummer. Nobody thinks of my needs. Nutrition has made my right eye really great but the left eye has triple vision I think. I'm getting real tired of it too I'd like to see the real Demi Moore stand up. Maybe they'll satisfy my detractors by implanting a pig's eye. I'm going to make an appt tomorrow and see er find out.

    • 2 years ago
  • tangibleparadox
    • 0
      tangibleparadox  

    • absolutely fantastic!!!

      "Much more needs to be learned about the safety of the treatment, its durability and other factors. But so far, improvement in vision has persisted as long as two years after a single treatment without evidence of significant adverse side effects."

      hopefully this proves to be a long-lasting procedure without unwanted side-effects... at least, it sounds like a step in the right direction. thanks for the post!

    • 2 years ago
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