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DNA-Mediated Gene Therapy in a Mouse Model of Limb Girdle Muscular Dystrophy 2B

Mutations in the gene for dysferlin cause a degenerative disorder of skeletal muscle known as limb girdle muscular dystrophy 2B. To achieve gene delivery of plasmids encoding dysferlin to hind limb muscles of dysferlin knockout mice, we used a vascular injection method that perfused naked plasmid DN...

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Bibliografiska uppgifter
I publikationen:Mol Ther Methods Clin Dev
Huvudupphovsmän: Ma, Julia, Pichavant, Christophe, du Bois, Haley, Bhakta, Mital, Calos, Michele P.
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2017
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC5684445/
https://ncbi.nlm.nih.gov/pubmed/29159199
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.10.005
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