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

eutralizing antibodies against both naturally occurring and injected AAV, but these antibodies did not prevent the injected gene from producing the desired enzyme." High is director of the Center for Cellular and Molecular Therapeutics (CCMT) at Children's Hospital, which manufactured the vector used in the current study and the previous human trial for LCA.

In the human trial for LCA reported last year, the CHOP/Penn researchers, led by Bennett, High and retina specialist Albert M. Maguire, M.D., injected the vector into only one eye in each of their 12 patients. Because the treatment was experimental, researchers left one eye untreated in the event of unexpected complications. After the subjects experienced partially restored eyesight in their treated eyes, many were eager to receive the same treatment in the other eye. The current study advances that possibility, and the research team is planning another clinical trial of LCA gene therapy, which may include some of the subjects from the first group.

Additionally, the results may set the stage for gene therapy in LCA patients who were excluded from the previous trial. Adopting a conservative approach, the researchers did not treat patients who already had neutralizing antibodies against AAV in their blood. As many as a quarter of all people may carry these antibodies by their teenage and young adult years. Fortunately, unlike other organs, both human and animal eyes are insulated from these circulating antibodies. (Co-author

SOURCE The Children's Hospital of Philadelphia
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