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• W2891493876 abstract "Duchenne muscular dystrophy (DMD) is a progressive, devastating disease of skeletal and cardiac muscles caused by loss of expression of the dystrophin protein (1). While the dystrophin gene and protein were discovered more than 30 y ago (2, 3), an effective treatment that can be administered to every patient, irrespective of their individual gene mutation, is still not available. Lessons and challenges about treating DMD have been learned over the years while testing various experimental therapies, including cell and gene replacement therapies (4, 5). One of the challenges faced by gene replacement techniques is the potential reaction of the immune system to a foreign protein. Indeed, previous reports have raised concerns over immune system reactions to transplanted donor cells (6), viral vector proteins, or dystrophin itself (7, 8). In the current study, Ho et al. (9) find that the immune system can be trained to accept foreign vectors and their encoded proteins through DNA vaccination (Fig. 1).Fig. 1. Improving gene therapy outcomes through immune tolerization. An adeno-associated virus serotype 6 (AAV6) vector encoding a miniaturized version of dystrophin (mDys) was delivered to a mouse model of Duchenne muscular dystrophy ( mdx mouse). The mice were subsequently treated weekly with an engineered plasmid in which all immunostimulatory CpG motifs had been replaced with immunosuppressive GpG motifs and encoded the same mDys gene. Control mice were treated with plasmid lacking the mDys gene or were injected with saline. Mice vaccinated with the engineered plasmid encoding the mDys gene showed improved muscle strength and reduced antibody-mediated immune responses to the dystrophin protein and AAV6 vector, relative to control animals.Conceptually, gene therapy—the transfer of a “good” copy of a mutated or missing gene into a recipient cell—is the most direct way to achieve correction of many genetic disorders. It relies on … [↵][1]1To whom correspondence should be addressed. Email: emanuela.gussoni{at}enders.tch.harvard.edu. [1]: #xref-corresp-1-1" @default.
• W2891493876 created "2018-09-27" @default.
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• W2891493876 date "2018-09-06" @default.
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• W2891493876 title "Building immune tolerance through DNA vaccination" @default.
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• W2891493876 doi "https://doi.org/10.1073/pnas.1813461115" @default.
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