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Posted On : 01 Jan 2017
is there any specific treatment for lebers congenital amaurosis in india.if so where and whom to contact
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Expert Answer :
Gene therapy treatments are still in the trial phase but there have been successes. The results of a phase 1 trial conducted, by the University of Pennsylvania and Children’s Hospital of Philadelphia and published in 2009, showed sustained improvement in 12 subjects {ages 8 to 44} with RPE65-associated LCA after treatment with AAV2-hRPE65v2, a gene replacement therapy.
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