AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy - Wikidata - awgldk - TriplyDB

AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

http://www.wikidata.org/entity/Q35568525

about

Facioscapulohumeral dystrophy: the path to consensus on pathophysiology A long ncRNA links copy number variation to a polycomb/trithorax epigenetic switch in FSHD muscular dystrophy Targeting mRNA for the treatment of facioscapulohumeral muscular dystrophy Novel contrasting and labeling procedures for correlative microscopy of thawed cryosections.Influence of immune responses in gene/stem cell therapies for muscular dystrophies.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies.Systemic RNAi delivery to the muscles of ROSA26 mice reduces lacZ expression Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy.Efficient in vitro siRNA delivery and intramuscular gene silencing using PEG-modified PAMAM dendrimers.RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy.Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9.Generation of isogenic D4Z4 contracted and noncontracted immortal muscle cell clones from a mosaic patient: a cellular model for FSHD Animal models of muscular dystrophy Distribution of myosin heavy chain isoforms in muscular dystrophy: insights into disease pathology.Morpholino treatment improves muscle function and pathology of Pitx1 transgenic mice.Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies.Gene-based therapies of neuromuscular disorders: an update and the pivotal role of patient organizations in their discovery and implementation.Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.Systemic delivery of adeno-associated viral vectors.Genome engineering: a new approach to gene therapy for neuromuscular disorders.Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.Facioscapulohumeral Muscular Dystrophy.Facioscapulohumeral dystrophy: Activating an early embryonic transcriptional program in human skeletal muscle.

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P2860

AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

http://www.wikidata.org/entity/Q35568525

description

2011 nî lūn-bûn

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2011年の論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年论文

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2011年论文

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2011年论文

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name

AAV6-mediated systemic shRNA d ...... pulohumeral muscular dystrophy

@ast

AAV6-mediated systemic shRNA d ...... pulohumeral muscular dystrophy

@en

type

label

AAV6-mediated systemic shRNA d ...... pulohumeral muscular dystrophy

@ast

AAV6-mediated systemic shRNA d ...... pulohumeral muscular dystrophy

@en

prefLabel

AAV6-mediated systemic shRNA d ...... pulohumeral muscular dystrophy

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AAV6-mediated systemic shRNA d ...... pulohumeral muscular dystrophy

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Molecular Therapy

P1476

AAV6-mediated systemic shRNA d ...... pulohumeral muscular dystrophy

@en

P2093

Alessandro Nonis

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Clelia Di Serio

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Francesca Sanvito

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Jessica Wei

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Joel R Chamberlain

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Sergia Bortolanza

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Simona Maciotta

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P2860

Facioscapulohumeral dystrophy: incomplete suppression of a retrotransposed gene

DUX4, a candidate gene of facioscapulohumeral muscular dystrophy, encodes a transcriptional activator of PITX1

Short GCG expansions in the PABP2 gene cause oculopharyngeal muscular dystrophy

The DUX4 gene at the FSHD1A locus encodes a pro-apoptotic protein

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Targeted deletion of Dicer in the heart leads to dilated cardiomyopathy and heart failure

FRG1P is localised in the nucleolus, Cajal bodies, and speckles

Systematic RNAi studies on the role of Sp/KLF factors in globin gene expression and erythroid differentiation

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

Nucleotide sequence of the partially deleted D4Z4 locus in a patient with FSHD identifies a putative gene within each 3.3 kb element

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2055-2064

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scholarly article

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10.1038/MT.2011.153

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11

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19

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Davide Gabellini

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2011-08-09T00:00:00Z

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51559492

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21829175

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facioscapulohumeral muscular dystrophy

P932

3222524

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