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

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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Main Authors: Florian Barthélémy (Author), Richard T. Wang (Author), Christopher Hsu (Author), Emilie D. Douine (Author), Eugene E. Marcantonio (Author), Stanley F. Nelson (Author), M. Carrie Miceli (Author)
Format: Book
Published: Elsevier, 2019-12-01T00:00:00Z.
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3rd Floor Main Library

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