Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
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Open Access
- 25 July 2011
- journal article
- research article
- Published by Elsevier in The Lancet
- Vol. 378 (9791) , 595-605
- https://doi.org/10.1016/s0140-6736(11)60756-3
Abstract
No abstract availableKeywords
Funding Information
- Great Ormond Street Hospital Children's Charity. Newcastle University, UCL, and Oxford are partners in TREAT-NMD ((EC036825))
This publication has 34 references indexed in Scilit:
- One-year Treatment of Morpholino Antisense Oligomer Improves Skeletal and Cardiac Muscle Functions in Dystrophic mdx MiceMolecular Therapy, 2011
- Chronic Systemic Therapy With Low-dose Morpholino Oligomers Ameliorates the Pathology and Normalizes Locomotor Behavior in mdx MiceMolecular Therapy, 2011
- Muscle histology vs MRI in Duchenne muscular dystrophyNeurology, 2011
- In-frame Dystrophin Following Exon 51-Skipping Improves Muscle Pathology and Function in the Exon 52–Deficient mdx MouseMolecular Therapy, 2010
- Exon‐skipped dystrophins for treatment of Duchenne muscular dystrophy: Mass spectrometry mapping of most exons and cooperative domain designs based on single molecule mechanicsCytoskeleton, 2010
- Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept studyThe Lancet Neurology, 2009
- Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophyJournal of Clinical Investigation, 2009
- Sarcolemma-localized nNOS is required to maintain activity after mild exerciseNature, 2008
- Grandpa and I have Dystrophinopathy?: Approach to Asymptomatic HyperCKemiaPediatric Neurology, 2006
- Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotideThe Journal of Gene Medicine, 2005