Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice.
about
FUS stimulates microRNA biogenesis by facilitating co-transcriptional Drosha recruitmentProgress toward therapy with antisense-mediated splicing modulationThe mdx mouse model as a surrogate for Duchenne muscular dystrophyDelivery is key: lessons learnt from developing splice-switching antisense therapiesGene-mediated restoration of normal myofiber elasticity in dystrophic muscles.Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx miceThe status of exon skipping as a therapeutic approach to duchenne muscular dystrophyThe genomic architecture of sporadic heart failureIntratumoral decorin gene delivery by AAV vector inhibits brain glioblastomas and prolongs survival of animals by inducing cell differentiation.MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy.Emerging strategies for cell and gene therapy of the muscular dystrophiesAAV vectors for RNA-based modulation of gene expression.Cell based therapy for Duchenne muscular dystrophy.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs.AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping.Mutation-adapted U1 snRNA corrects a splicing error of the dopa decarboxylase gene.Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations.Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approachesmiRNAs as serum biomarkers for Duchenne muscular dystrophy.The pathology of pre-mRNA splicing: a meeting in the Italian Alps. Workshop on pre-mRNA processing and disease.
P2860
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P2860
Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice.
description
2006 nî lūn-bûn
@nan
2006年の論文
@ja
2006年論文
@yue
2006年論文
@zh-hant
2006年論文
@zh-hk
2006年論文
@zh-mo
2006年論文
@zh-tw
2006年论文
@wuu
2006年论文
@zh
2006年论文
@zh-cn
name
Chimeric adeno-associated viru ...... y local treatment of mdx mice.
@en
Chimeric adeno-associated viru ...... y local treatment of mdx mice.
@nl
type
label
Chimeric adeno-associated viru ...... y local treatment of mdx mice.
@en
Chimeric adeno-associated viru ...... y local treatment of mdx mice.
@nl
prefLabel
Chimeric adeno-associated viru ...... y local treatment of mdx mice.
@en
Chimeric adeno-associated viru ...... y local treatment of mdx mice.
@nl
P2093
P356
P1433
P1476
Chimeric adeno-associated viru ...... y local treatment of mdx mice.
@en
P2093
Alberto Auricchio
Antonio Musarò
Carmine Nicoletti
Fernanda Gabriella De Angelis
Giuseppe D'Antona
Irene Bozzoni
Mariacarmela Allocca
Olga Sthandier
Orietta Pansarasa
Roberto Bottinelli
P304
P356
10.1089/HUM.2006.17.565
P577
2006-05-01T00:00:00Z