Word: leber
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Dates: during 2000-2009
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...Born with Leber Congenital Amaurosis, a genetic disease that prevented her eyes from fully developing, Stevens has some vision, though not enough to distinguish the cars as they circle the track...
...patients have a faulty gene that gives them a form of Leber's Congenital Amaurocis, a major cause of congenital blindness. Born with limited sight, the patients are expected to become totally blind as they grow older. They have enough photoreceptor cells, but the cells don't work because one of the genes is a dud. Now Ali is firing functional versions of the faulty gene back into the photoreceptor cells, and the one-time procedure could permanently cure the blindness. Johnson's condition - a faulty RPE65 gene - is incredibly rare. But Ali says the procedure, if it works, could...