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Gene Therapy Transforms Eyesight Of 12 People With Rare Visual Defect
LATimes ^ | October 24th 2009

Posted on 10/24/2009 2:00:36 PM PDT by Steelfish

Gene Therapy Transforms Eyesight Of 12 People With Rare Visual Defect

A single injection in a patient's eye brings 'astounding' results. The findings may offer hope for those with macular degeneration and retinitis pigmentosa.

Thomas H. Maugh II October 24, 2009

Pennsylvania researchers using gene therapy have made significant improvements in vision in 12 patients with a rare inherited visual defect, a finding that suggests it may be possible to produce similar improvements in a much larger number of patients with retinitis pigmentosa and macular degeneration.

The team last year reported success with three adult patients, an achievement that was hailed as a major accomplishment for gene therapy. They have now treated an additional nine patients, including five children, and find that the best results are achieved in the youngest patients, whose defective retinal cells have not had time to die off.

The youngest patient, 9-year-old Corey Haas, was considered legally blind before the treatment began. He was confined largely to his house and driveway when playing, had immense difficulties in navigating an obstacle course and required special enlarging equipment for books and help in the classroom.

(Excerpt) Read more at latimes.com ...


TOPICS: Chit/Chat; Science
KEYWORDS: blind; blindness; eyedisease; eyesight; eyetreatment; lebers; macular; maculardegeneration

1 posted on 10/24/2009 2:00:36 PM PDT by Steelfish
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To: Steelfish

REMARKABLE!


2 posted on 10/24/2009 2:11:16 PM PDT by Doctor Don
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To: Steelfish

So is this the result of embryonic stem cell research? I’m betting no.


3 posted on 10/24/2009 2:19:16 PM PDT by Brilliant
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To: Brilliant
No mention of stem cells that I read but the popular press is nowhere to get detailed, reliable scientific information.

Looks good to me.

4 posted on 10/24/2009 2:29:15 PM PDT by muir_redwoods (Obama: The Fresh Prince of Bill Ayers)
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To: Steelfish

This is a great story. I help moderate a yahoo group about kids with hearing loss. A couple parents have kids who are deaf AND going blind. One of the moms has several kids (close in age) in the same situation. I sent them this link. Thank you for posting it.


5 posted on 10/24/2009 2:30:48 PM PDT by merry10
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To: Pharmboy; Coleus; neverdem; AdmSmith; Berosus; bigheadfred; Convert from ECUSA; dervish; ...

Thanks Steelfish.


6 posted on 10/25/2009 3:30:00 PM PDT by SunkenCiv (https://secure.freerepublic.com/donate/__Since Jan 3, 2004__Profile updated Monday, January 12, 2009)
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To: Steelfish

wow

cloned cells


7 posted on 10/25/2009 8:55:55 PM PDT by dervish (I never saw a wild thing sorry for itself)
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To: merry10

Merry10, your FR homepage is not very merry.


8 posted on 10/25/2009 9:01:41 PM PDT by Yardstick
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To: SunkenCiv

thanks, bfl


9 posted on 10/26/2009 12:14:00 AM PDT by neverdem (Xin loi minh oi)
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To: Steelfish; SunkenCiv
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
10 posted on 10/26/2009 12:35:48 AM PDT by neverdem (Xin loi minh oi)
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To: Yardstick

you are correct. I should clean it up.


11 posted on 10/30/2009 1:07:45 PM PDT by merry10
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