A Rocking Good Sunday
As temperatures reached triple digits here in Los Angeles, I retreated to a friend's pool to cool my heels and other parts. The heat so drained me of motivation, it was all I could do to make dinner and mix my adult beverage. But hey, who does anything worthwhile on a steamy Sunday? Turns out, scientists, that's who.
As evening fell and the hills burned, we made our way inside for a marathon session of Rockband (big ups! to my bandmates, the Clark sisters). At the same time, researchers in Philadelphia were publishing the astounding results of their work. 'What did they do?' you ask. Just a little cure for blindness, that's all.
It's really quite awesome. Of course I don't mean awesome like god because these researchers actually made the blind see. It's awesome because the research points to the efficacy of what would be a paradigm shifting technique. Not only were the results transformative for the patients, but also the successful use of the techniques being tested is great news for a whole field of treatment for a myriad of medical disorders.
The researchers used gene therapy on patients with Leber's congenital amaurosis, a congenital condition (usually autosomal recessive inheritance) that is estimated to be responsible for anywhere from 10% to 18% of congenital blindness.
The patients showed marked improvement from the treatment. This could be that proverbial shot in the arm to the ailing field of gene therapy. The practice, which - simply put - replaces a faulty gene in a patient with a functioning one, holds a great promise, but hasn't seen many great successes since research first began fifteen years ago. This study, published on Sunday in The New England Journal of Medicine, is being touted as a "major advance" for gene therapy.
My lazy Sunday self-loathing notwithstanding, I am so pleased by the news of the researchers' success. Further, I am glad there are some folks who can put their weekends to such good use. Although, in my own defense, I did score a 241 note streak and 100% for my guitar performance of REM's "Orange Crush."
Oh, and I checked the weather in Philadelphia on Sunday. It was cool and overcast. I bet I'd have done lots of productive stuff if I'd been there.
Tags: gene therapy, New England Journal of Medicine, Rockband







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2 Comments
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April 29, 2008 12:12 PM
nomad
"Maguire watched through a microscope as he and his team did the work. He operated in stocking feet because he had to control two of his tools, the vitrector and the microscope, with foot pedals. Each had a series of switches that he operated with his toes."
So perhaps the drums are the way to go next time?
I kid. Wow. This is really cool and exciting stuff. Thanks for the info.
April 29, 2008 7:06 PM
damon
Ahh, a quote from an article I linked to! Nomad, you warm my heart with your dedicated clicking-through, your inverting of my first name, and, of course, your understanding of the hand/foot-eye coordination training Rockband affords the dedicated user.
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