Gene Therapy

Data from University of Pennsylvania provide new insights into gene therapy

Published in Gene Therapy Weekly, November 5th, 2009

According to a study from the United States, "Human gene therapy with rAAV2-vector was performed for the RPE65 form of childhood blindness called Leber congenital amaurosis. In three contemporaneous studies by independent groups, the procedure was deemed safe and there was evidence of visual gain in the short term."

"At 12 months after treatment, our young adult subjects remained healthy and without vector-related serious adverse events. Results of immunological assays to identify reaction to AAV serotype 2 capsid were unchanged from baseline measurements. Results of clinical eye examinations of study and control eyes, including visual acuities and central retinal...

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Source: Gene Therapy Weekly (2009-11-05)

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