Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
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Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyRescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in miceMetabolic dysfunction and altered mitochondrial dynamics in the utrophin-dystrophin deficient mouse model of duchenne muscular dystrophySelective release of muscle-specific, extracellular microRNAs during myogenic differentiationLow dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout micePrevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 SplicingTherapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications.High throughput screening in duchenne muscular dystrophy: from drug discovery to functional genomicsSkeletal muscle fibrosis in the mdx/utrn+/- mouse validates its suitability as a murine model of Duchenne muscular dystrophy.Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.Autonomic Modulation in Duchenne Muscular Dystrophy during a Computer Task: A Prospective Control Trial.Muscle-specific microRNAs as biomarkers of Duchenne Muscular Dystrophy progression and response to therapies2015 William Allan Award.Prospect of gene therapy for cardiomyopathy in hereditary muscular dystrophy.Dystrophin restoration therapy improves both the reduced excitability and the force drop induced by lengthening contractions in dystrophic mdx skeletal muscle.Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnoverRepair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.Detection and quantification of extracellular microRNAs in murine biofluids.Nanotherapy for Duchenne muscular dystrophy.AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse ModelVEGF induces stress fiber formation in fibroblasts isolated from dystrophic muscle.Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD
P2860
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P2860
Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
description
2012 nî lūn-bûn
@nan
2012 թուականի Մարտին հրատարակուած գիտական յօդուած
@hyw
2012 թվականի մարտին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
Rescue of severely affected dy ...... U7snRNA-mediated exon skipping
@ast
Rescue of severely affected dy ...... U7snRNA-mediated exon skipping
@en
type
label
Rescue of severely affected dy ...... U7snRNA-mediated exon skipping
@ast
Rescue of severely affected dy ...... U7snRNA-mediated exon skipping
@en
prefLabel
Rescue of severely affected dy ...... U7snRNA-mediated exon skipping
@ast
Rescue of severely affected dy ...... U7snRNA-mediated exon skipping
@en
P2093
P2860
P356
P1476
Rescue of severely affected dy ...... U7snRNA-mediated exon skipping
@en
P2093
Arran Babbs
Dave Powell
Jordan Wright
Kay E Davies
Luis Garcia
Vivienne Wilkins
P2860
P304
P356
10.1093/HMG/DDS082
P577
2012-03-02T00:00:00Z