Sci Rep. 2017 Dec;7(1):79. doi: 10.1038/s41598-017-00152-5. Epub 2017 Mar 6.
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In this work, the scientists have demonstrated that lentiviral vectors can package and deliver inserts of a similar size to dystrophin. They report a novel approach for delivering large transgenes in lentiviruses, in which they demonstrate proof-of-concept for a 'template-switching' lentiviral vector that harnesses recombination events during reverse-transcription. Their results shown gene therapy with this vector by restoring dystrophin expression in DMD myoblasts, where dystrophin was expressed at the sarcolemma of myotubes after myogenic differentiation.
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In this work, the scientists have demonstrated that lentiviral vectors can package and deliver inserts of a similar size to dystrophin. They report a novel approach for delivering large transgenes in lentiviruses, in which they demonstrate proof-of-concept for a 'template-switching' lentiviral vector that harnesses recombination events during reverse-transcription. Their results shown gene therapy with this vector by restoring dystrophin expression in DMD myoblasts, where dystrophin was expressed at the sarcolemma of myotubes after myogenic differentiation.