Bobby Gaspar: "Translational research in the ex vivo gene therapy of monogenic diseases" Educational Day* at ESGCT Conference in Madrid. "Translational research in the ex vivo gene therapy of monogenic diseases". Bobby Gaspar, Professor of Paedi... By: European Society for Gene and Cell Therapy … Continue reading →

Chiara Bonini: "Bases of Gene Therapy in leukemias" Chiara Bonini, dept. of Experimental Hematology at Fondazione San Raffaele del Monte Tabor - Milano, talks about "Bases of Gene Therapy in leukemias". http:/... By: European Society for Gene and Cell Therapy … Continue reading →

Contact Information Available for logged-in reporters only Newswise (MEMPHIS, Tenn. November 19, 2014) Gene therapy developed at St. Jude Childrens Research Hospital, University College London (UCL) and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported. The results appear in the November 20 edition of The New England Journal of Medicine. In this study, participants received one of three doses of gene therapy that used a modified adeno-associated virus (AAV) 8 as the vector to deliver the genetic material for making Factor IX. The vector was administered as a single infusion into a peripheral vein in the arm of each participant. Factor IX levels rose in all 10 men with severe hemophilia B following gene therapy and have remained stable for more than four years. Overall, episodes of spontaneous bleeding declined 90 percent. Use of Factor IX replacement therapy dropped about 92 percent in the first 12 months after the treatment with the investigational therapy. In the six participants who received the highest gene therapy dose, levels of … Continue reading →

LOS ANGELES (KABC) -- In bright daylight, 10-year-old Mark Devoe has no trouble seeing his friends. But inside or even in the shade, Mark's eyes sometimes don't work. "I have trouble seeing like, trees, when the road ends, and when there's like a drop there," said Mark. At age 6, Mark's doctors diagnosed him with the genetic condition choroideremia, which causes people to progressively lose vision until they are completely blind. "I don't know what it's like to live in darkness, but I've seen it," said Susan Devoe, Mark's mother. Susan is a carrier of the blindness gene. Mark's grandfather has the condition. "Watching my father go blind was devastating," said Susan. "I was a little girl. You know, you count on daddy to do things, and daddy couldn't do them." Dr. Jean Bennett is one of two U.S. researchers preparing to test a gene therapy for choroideremia in humans. "I think gene therapy holds a huge promise for developing treatments for blinding diseases," said Bennett. Researchers will use a virus carrying a small choroideremia gene and inject the virus just under the retina. The gene should begin to work in a few weeks. Go here to see the original: … Continue reading →