Liver-directed lentiviral gene therapy is an effective treatment for hemophilia B in dogs, according to Alessio Cantore, Ph.D., and his associates.
The investigators found that all of the dogs administered the treatment were still alive 5 years after the therapy. Spontaneous bleeding was cut down significantly from before the therapy began. No significant side effects were seen in any of the dogs, and no lentiviral vector DNA was found in blood samples, proving long-term effectiveness of vector-transduced cells.
“If we extrapolate the observed dose response in dogs to humans, the current lentiviral vector–manufacturing capacity could support the treatment of adult patients with hemophilia” if a hyperfunctional IX transgene factor were used, Dr. Cantore and his associates wrote. “Lentiviral vectors may complement other available vectors to address some of the outstanding challenges posed by liver gene therapy for the treatment of hemophilia and conceivably other diseases.”
Read the full study in Science Translational Medicine (doi: 10.1126/scitranslmed.aaa1405).