Exon skipping and translation in patients with frameshift deletions in the dystrophin gene.
about
The pathobiology of splicingIdentification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screeningPrevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.Octa-guanidine morpholino restores dystrophin expression in cardiac and skeletal muscles and ameliorates pathology in dystrophic mdx mice.Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.Modulation of in vitro splicing of the upstream intron by modifying an intra-exon sequence which is deleted from the dystrophin gene in dystrophin Kobe.Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko miceOne-year treatment of morpholino antisense oligomer improves skeletal and cardiac muscle functions in dystrophic mdx mice.Induction of revertant fibres in the mdx mouse using antisense oligonucleotidesWhen cells stop making sense: effects of nonsense codons on RNA metabolism in vertebrate cells.Therapeutic approaches to muscular dystrophy.Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through.The regulation of splice-site selection, and its role in human diseaseGerm-line p53 mutations in 15 families with Li-Fraumeni syndrome.Frameshift deletions of exons 3-7 and revertant fibers in Duchenne muscular dystrophy: mechanisms of dystrophin production.Effect of adopting a new histological grading system of acute rejection after heart transplantation.Mammalian nonsense codons can be cis effectors of nuclear mRNA half-life.Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer.Novel compounds for the treatment of Duchenne muscular dystrophy: emerging therapeutic agentsEmerging strategies for cell and gene therapy of the muscular dystrophiesMutation types and aging differently affect revertant fiber expansion in dystrophic mdx and mdx52 mice.Antisense suppression of donor splice site mutations in the dystrophin gene transcript.Transcription of the dystrophin gene in normal tissues and in skeletal muscle of a family with X-linked dilated cardiomyopathy.Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.Defects in RNA splicing and the consequence of shortened translational reading frames.Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin.Genetic modifiers of Duchenne and facioscapulohumeral muscular dystrophies.
P2860
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P2860
Exon skipping and translation in patients with frameshift deletions in the dystrophin gene.
description
1993 nî lūn-bûn
@nan
1993年の論文
@ja
1993年論文
@yue
1993年論文
@zh-hant
1993年論文
@zh-hk
1993年論文
@zh-mo
1993年論文
@zh-tw
1993年论文
@wuu
1993年论文
@zh
1993年论文
@zh-cn
name
Exon skipping and translation ...... etions in the dystrophin gene.
@en
type
label
Exon skipping and translation ...... etions in the dystrophin gene.
@en
prefLabel
Exon skipping and translation ...... etions in the dystrophin gene.
@en
P2093
P2860
P1476
Exon skipping and translation ...... letions in the dystrophin gene
@en
P2093
P2860
P304
P407
P577
1993-11-01T00:00:00Z