as mentioned in ibtimes
Promising pig study suggests gene therapy can treat metabolic liver disease
Promising pig study suggests gene therapy can treat metabolic liver disease
Gene therapy has proved to be a promising treatment against metabolic liver disease in animal trials.Pigs transplanted with gene-corrected liver cells avoided many complications associated with the illness.Today, patients who suffer from inherited metabolic liver disease have little option other than liver transplantation as they often become resistant to current treatments.
moreover from eurekalert
New study shows long-term safety of gene therapy in Parkinson's disease
New study shows long-term safety of gene therapy in Parkinson's disease
New Rochelle, NY, July 27, 2016--New safety data from a study of patients with advanced Parkinson's disease five years after gene transfer-mediated delivery of the neuroprotective factor neurturin directly to patients' brains reveal no serious adverse events related to the treatment.The encouraging long-term safety profile of the surgically administered adeno-associated virus (AAV2)-neurturin gene therapy is described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.The article is part of a special issue on CNS disorders and is available free for download on the Human Gene Therapy website until August 27, 2016.
besides fiercebiotech
Sangamo gene therapy for hemophilia shows positive results in mice, monkeys
Sangamo gene therapy for hemophilia shows positive results in mice, monkeys
Gene editing pioneer Sangamo BioSciences ($SGMO) has its sights set on curing hemophilia--a rare disorder in which the blood doesn't clot normally.In new data presented at the World Federation of Hemophilia's annual meeting this week in Orlando, FL, the company's gene therapy significantly increased an essential blood-clotting protein in animals that's deficient in individuals with hemophilia A.The optimistic preclinical results mean Sangamo plans to move the investigational gene therapy into the clinic.
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