The experimental gene therapy which CURED once-blind America's Got Talent semi-finalist Christian Guardino is now set for FDA approval

Mail Online | 10/9/2017 | Associated Press; Reporter
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For Christian Guardino, a senior at Patchogue-Medford High School on Long Island, the most remarkable part about performing on America's Got Talent a day before his 17th birthday earlier this year wasn't winning the golden buzzer that showered gold confetti on him and sent him into further competition.

It was seeing the confetti thanks to his gene therapy several years ago.

Stage - Judges

'I walked out on that stage all by myself,' he said. 'I saw the judges. It was incredible.'

His mother, Beth Guardino, said the judges didn't know about Christian's blindness and gene therapy until after his audition.

Treatment - Dark - Life - Christian - Way

Before treatment, 'it was dark, life without light,' Christian said. 'I found a way to work through it, to cope with it, and that was music.'

He had mere glimmers of vision and was destined to lose even that because of an inherited eye disease with no treatment or cure.

Treatment - Things - Stars - Fireworks - Moon

Since treatment, 'I've been able to see the most incredible things. I'm able to see stars, I'm able to see fireworks, snow falling,' he said. His favorite? 'The moon. Most definitely. I'm a huge astronomy fan.'

Now, other children may get to feel the same euphoria.

Thursday - US - Food - Drug - Administration

On Thursday, U.S. Food and Drug Administration advisers will consider whether to recommend approval of a gene therapy that improved vision for these three youths and some others with hereditary blindness.

It would be the first gene therapy in the U.S. for an inherited disease, and the first in which a corrective gene is given directly to a patient. Only one gene therapy is sold in the U.S. now, a cancer treatment approved in August that engineers patients' blood cells in the lab.



The therapy has wider implications but was tested for Leber congenital amaurosis, or LCA, caused by flaws in a gene called RPE65. Those with it can't make...
(Excerpt) Read more at: Mail Online
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