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Gene Therapy Improves Vision in Patients with Congenital Retinal Disease
Date:4/27/2008

he Second University of Naples, an institution with long-standing experience in collecting and studying patients with inherited retinal diseases, under the supervision of Francesca Simonelli, M.D.

Testing continued over a period of six months following the gene therapy vector administration. One patient was better able to navigate an obstacle course compared to before the injection. The patients also had less nystagmus, an involuntary movement of the eyes that is common in LCA. In the patient who experienced better vision even in the uninjected eye, the researchers suggest that the reduced nystagmus benefited both eyes.

"The current clinical trial will continue with more patients and with ongoing follow-up to monitor results," said Bennett. "We expect improvements to be more pronounced if treatment occurs in childhood, before the disease progresses."

"This result is important for the entire field of gene therapy," notes High, a past president of the American Society of Gene Therapy. "Gene transfer has been in clinical trials for over 15 years now, and although it has an excellent safety record, examples of therapeutic effect are still relatively few. The results in this study provide objective evidence of improvement in the ability to perceive light, and thus lay the groundwork for future studies in this and other retinal disorders," said High.

The pace of moving from pre-clinical discoveries into clinical trials has typically been slow in the field of gene therapy due to the breadth of expertise required, ranging from in-depth knowledge of the disorder to detailed understanding of vector design, manufacture, and pre-clinical evaluation. The complexities of regulatory oversight at both the federal and local levels also present challenges. Through the Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia has developed concentrated expertise and substantial resources to facilitate the "bench to beds
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SOURCE The Children's Hospital of Philadelphia; University ofPennsylvania
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