Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy

In gene therapy for Duchenne muscular dystrophy there are two potential immunological obstacles. An individual with Duchenne muscular dystrophy has a genetic mutation in dystrophin, and therefore the wild-type protein is “foreign,” and thus potentially immunogenic. The adeno-associated virus serotyp...

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Tallennettuna:
Bibliografiset tiedot
Julkaisussa:Proc Natl Acad Sci U S A
Päätekijät: Ho, Peggy P., Lahey, Lauren J., Mourkioti, Foteini, Kraft, Peggy E., Filareto, Antonio, Brandt, Moritz, Magnusson, Klas E. G., Finn, Eric E., Chamberlain, Jeffrey S., Robinson, William H., Blau, Helen M., Steinman, Lawrence
Aineistotyyppi: Artigo
Kieli:Inglês
Julkaistu: National Academy of Sciences 2018
Aiheet:
PNAS Plus
Linkit:https://ncbi.nlm.nih.gov/pmc/articles/PMC6166850/
https://ncbi.nlm.nih.gov/pubmed/30181272
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1808648115
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