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 study - Wikidata - wikimedia - TriplyDB

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 study

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 study

http://www.wikidata.org/entity/Q24643018

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Molecular and cell-based therapies for muscle degenerations: a road under construction Drug treatment of Duchenne muscular dystrophy: available evidence and perspectives 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 Current and emerging treatment strategies for Duchenne muscular dystrophy Duchenne Muscular Dystrophy: From Diagnosis to Therapy Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophies New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicals Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery Antisense oligonucleotides: treating neurodegeneration at the level of RNA Potential molecular targeting of splice variants for cancer treatment Splicing therapy for neuromuscular disease Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells.A splice site mutation in laminin-α2 results in a severe muscular dystrophy and growth abnormalities in zebrafish Nutraceuticals and Their Potential to Treat Duchenne Muscular Dystrophy: Separating the Credible from the Conjecture Advances in gene therapy for muscular dystrophies Current understanding of molecular pathology and treatment of cardiomyopathy in duchenne muscular dystrophy Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient Targeted skipping of human dystrophin exons in transgenic mouse model systemically for antisense drug development Nanobiopolymer for direct targeting and inhibition of EGFR expression in triple negative breast cancer Guidance in social and ethical issues related to clinical, diagnostic care and novel therapies for hereditary neuromuscular rare diseases: "translating" the translational A sensitive, reproducible and objective immunofluorescence analysis method of dystrophin in individual fibers in samples from patients with duchenne muscular dystrophy Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method.Enhancement of Blood-Brain Barrier Permeability and Delivery of Antisense Oligonucleotides or Plasmid DNA to the Brain by the Combination of Bubble Liposomes and High-Intensity Focused Ultrasound.Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping Skeletal muscles of ambulant children with Duchenne muscular dystrophy: validation of multicenter study of evaluation with MR imaging and MR spectroscopy.The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.Rapamycin nanoparticles target defective autophagy in muscular dystrophy to enhance both strength and cardiac function Comparison of dixon and T1-weighted MR methods to assess the degree of fat infiltration in duchenne muscular dystrophy patients.Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo.Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor.Exon exchange approach to repair Duchenne dystrophin transcripts Dystrophic Cardiomyopathy-Potential Role of Calcium in Pathogenesis, Treatment and Novel Therapies Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.What Can We Learn From Clinical Trials of Exon Skipping for DMD?Evaluation of Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked polyethylenimine as antisense morpholino oligomer delivery vehicle in cell culture and dystrophic mdx mice Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.

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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 study

http://www.wikidata.org/entity/Q24643018

description

2009 nî lūn-bûn

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2009 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած

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2009 թվականի հոտեմբերին հրատարակված գիտական հոդված

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2009年の論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年論文

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2009年论文

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Lancet Neurology

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Caroline McCulley

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Caroline Sewry

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P2860

Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide.

Dystrophin and mutations: one gene, several proteins, multiple phenotypes.

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Somatic reversion/suppression in Duchenne muscular dystrophy (DMD): evidence supporting a frame-restoring mechanism in rare dystrophin-positive fibers.

The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion

Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion.

Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy.

Muscle MRI in inherited neuromuscular disorders: past, present, and future.

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Francesco Muntoni

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