The current study is sponsored by the Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia, directed by Katherine A. High, M.D. High, a study leader and an Investigator of the Howard Hughes Medical Institute, has been a pioneer in translational and clinical studies of gene therapy for genetic disease, and in 2005 initiated a collaboration with Bennett and her group to translate their exciting animal findings into a clinical study.
The scientists used a vector, a genetically engineered adeno-associated virus, to carry a normal version of the gene, called RPE65, that is mutated in one form of LCA. Three patients, ages 19, 26 and 26, received the gene therapy via a surgical procedure performed by Maguire between October 2007 and January 2008 at The Children's Hospital of Philadelphia, where the gene vector was manufactured at the hospital's Center for Cellular and Molecular Therapeutics (CCMT).
Starting two weeks after the injections, all three patients reported improved vision in the injected eye. "Standard vision tests showed significantly improved vision in the patients," said Alberto Auricchio, M.D., a study leader from the Telethon Institute of Genetics and Medicine and University of Naples Federico II. The researchers also reported that each injected eye became approximately three times more sensitive to light, and each was improved compared to the uninjected, previously better functioning eye.
The LCA gene therapy vector showed no signs of causing inflammation in the retina or other toxic side effects. One of the three patients had an adverse event, a hole in the retina that did not affect eyesight and may have been surgery-related, rather than related to biological effects of the therapeutic gene or the vector used to carry it.
The patients enrolled in the study to date were identified at the
Department of Ophthalmology at t
|SOURCE The Children's Hospital of Philadelphia; University ofPennsylvania|
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