Gene Therapy (2009) for Leber's Disease


In a 2009 Phase I clinical trial, researchers injected a benign virus carrying a connected copy of an essential gene into a teenage boy's retina. This patient had lost vision due to a genetic disorder called "Leber's Congenital Amaurosis (LGA).

The result was improved vision as the gene therapy enabled the body to make new rods and cones (which normally are irreplaceable once lost).

Other young patients given the same procedure also were able to see better. All the children that underwent this procedure gained enough vision to walk independently.

Published: The Lancet, 2009, by Albert M Maguire MD and others, University of Pennsylvania School of Medicine

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