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Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models

Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression, and slowing disease progression in animal models. In humans with Duchenne muscular dystrophy treated with AOs, low levels of dystrophin have been in...

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Detalles Bibliográficos
Autores principales:	Barthélémy, Florian, Wang, Richard T., Hsu, Christopher, Douine, Emilie D., Marcantonio, Eugene E., Nelson, Stanley F., Miceli, M. Carrie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2019
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6838898/ https://www.ncbi.nlm.nih.gov/pubmed/31678734 http://dx.doi.org/10.1016/j.omtn.2019.09.020

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