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Second Dose of Gene Therapy for Inherited Blindness Proves Safe in Animal Studies
Date:3/3/2010

t Children's Hospital.

"We designed this study to investigate the immunological consequences of administering the gene therapy injection to the second eye after treating the first one," said corresponding author Jean Bennett, M.D., Ph.D., F.M. Kirby professor of Ophthalmology at the University of Pennsylvania School of Medicine. "The good news is that in animals, the second injection, like the first, is benign."

As in the human trials of this gene therapy, the researchers packaged a normal version of the gene that is missing in LCA inside a genetically engineered vector, adeno-associated virus (AAV). The vector delivers the gene to cells in the retina, where the gene produces an enzyme that restores light receptors. Although the virus used does not cause human disease, it previously set off an immune response that cut short the initial benefits of gene therapy, notably in a 2002 human trial of gene therapy for the bleeding disorder hemophilia.

"Our current study in large animals provides encouraging indications that immune responses will not interfere with human gene therapy in both eyes," said co-author Katherine A. High, M.D., a pioneer in gene therapy who helped lead the hemophilia trial. "Like humans, monkeys generate n
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SOURCE The Children's Hospital of Philadelphia
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