Exon exchange approach to repair Duchenne dystrophin transcripts
about
mRNA trans-splicing in gene therapy for genetic diseasesRepair of Mybpc3 mRNA by 5'-trans-splicing in a Mouse Model of Hypertrophic CardiomyopathyMYBPC3 in hypertrophic cardiomyopathy: from mutation identification to RNA-based correctionRepair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa.Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy.Reprogramming the Dynamin 2 mRNA by Spliceosome-mediated RNA Trans-splicing.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.A novel screening system improves genetic correction by internal exon replacement.Cis-splicing and translation of the pre-trans-splicing molecule combine with efficiency in spliceosome-mediated RNA trans-splicingCorrection of the Middle Eastern M712T mutation causing GNE myopathy by trans-splicing.Considerations for a Successful RNA Trans-splicing Repair of Genetic DisordersDesigning Efficient Double RNA trans-Splicing Molecules for Targeted RNA Repair.Evaluation of MYBPC3 trans-Splicing and Gene Replacement as Therapeutic Options in Human iPSC-Derived Cardiomyocytes.Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approachesGene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy.RNA Trans-Splicing Modulation via Antisense Molecule Interference.Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.
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P2860
Exon exchange approach to repair Duchenne dystrophin transcripts
description
2010 nî lūn-bûn
@nan
2010 թուականի Մայիսին հրատարակուած գիտական յօդուած
@hyw
2010 թվականի մայիսին հրատարակված գիտական հոդված
@hy
2010年の論文
@ja
2010年論文
@yue
2010年論文
@zh-hant
2010年論文
@zh-hk
2010年論文
@zh-mo
2010年論文
@zh-tw
2010年论文
@wuu
name
Exon exchange approach to repair Duchenne dystrophin transcripts
@ast
Exon exchange approach to repair Duchenne dystrophin transcripts
@en
type
label
Exon exchange approach to repair Duchenne dystrophin transcripts
@ast
Exon exchange approach to repair Duchenne dystrophin transcripts
@en
prefLabel
Exon exchange approach to repair Duchenne dystrophin transcripts
@ast
Exon exchange approach to repair Duchenne dystrophin transcripts
@en
P2860
P1433
P1476
Exon exchange approach to repair Duchenne dystrophin transcripts
@en
P2093
Luis Garcia
Maëva Le Hir
P2860
P304
P356
10.1371/JOURNAL.PONE.0010894
P407
P577
2010-05-28T00:00:00Z