Gene Therapy for Macular Degeneration

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Genetic Engineering News reports on a small trial of a new gene therapy: "An adenoviral-based vector containing the gene for human pigment epithelium-derived factor (PEDF) showed evidence of being able to stop disease progression when injected directly into the eyes of patients with neovascular age-related macular degeneration, according to the results of a phase I clinical trial." The positive effects were modest - at this stage in the development of gene therapies, scientists are delighted to obtain modest benefits with no accompanying issues or problem. "The results from the Campochiaro et al. study are extremely encouraging as there were no serious adverse events or dose-limiting toxicities through the highest dose."

Link: http://www.genengnews.com/news/bnitem.aspx?name=1144877XSL_NEWSML_TO_NEWSML.xml