Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.
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mRNA trans-splicing in gene therapy for genetic diseasesChimeric RNAs in cancer and normal physiology.New high-technology products for the treatment of haemophilia.Exon exchange approach to repair Duchenne dystrophin transcriptsExpression of Herpes Simplex Virus Thymidine Kinase/Ganciclovir by RNA Trans-Splicing Induces Selective Killing of HIV-Producing Cells.K14 mRNA reprogramming for dominant epidermolysis bullosa simplex.Reprogramming of tau alternative splicing by spliceosome-mediated RNA trans-splicing: implications for tauopathies.5' exon replacement and repair by spliceosome-mediated RNA trans-splicingIdentification of a splicing enhancer in MLH1 using COMPARE, a new assay for determination of relative RNA splicing efficienciesCurrent status of gene therapy for inherited lung diseasesType XVII collagen gene mutations in junctional epidermolysis bullosa and prospects for gene therapy.Trans-splicing repair of mutant p53 suppresses the growth of hepatocellular carcinoma cells in vitro and in vivo.Targeted genetic repair: an emerging approach to genetic therapy.Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicingFunctional repair of p53 mutation in colorectal cancer cells using trans-splicing.Repair of rhodopsin mRNA by spliceosome-mediated RNA trans-splicing: a new approach for autosomal dominant retinitis pigmentosa.The cystic fibrosis gene: a molecular genetic perspectiveModulating the expression of disease genes with RNA-based therapy.Replacement of huntingtin exon 1 by trans-splicing.Gene therapy for muscular dystrophy: current progress and future prospects.New developments in the use of gene therapy to treat Duchenne muscular dystrophy.Construction and validation of an RNA trans-splicing molecule suitable to repair a large number of COL7A1 mutations.Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites.Targeting Splicing in the Treatment of Human DiseaseFunctional cystic fibrosis transmembrane conductance regulator expression in cystic fibrosis airway epithelial cells by AAV6.2-mediated segmental trans-splicingUsing 5'-PTMs to repair mutant beta-globin transcripts.Adeno-Associated Virus (AAV) gene therapy for cystic fibrosis: current barriers and recent developments.Development of spliceosome-mediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases.Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells.Optimization of trans-Splicing for Huntington's Disease RNA Therapy.Naturally occurring heterologous trans-splicing of adenovirus RNA with host cellular transcripts during infection.Correction of dystrophia myotonica type 1 pre-mRNA transcripts by artificial trans-splicing.Molecular barcoding of viral vectors enables mapping and optimization of mRNA trans-splicing.Gene Therapy via Trans-Splicing for LMNA-Related Congenital Muscular Dystrophy.Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.Centronuclear myopathies under attack: A plethora of therapeutic targets
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P2860
Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.
description
2000 nî lūn-bûn
@nan
2000年の論文
@ja
2000年学术文章
@wuu
2000年学术文章
@zh-cn
2000年学术文章
@zh-hans
2000年学术文章
@zh-my
2000年学术文章
@zh-sg
2000年學術文章
@yue
2000年學術文章
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2000年學術文章
@zh-hant
name
Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.
@en
type
label
Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.
@en
prefLabel
Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.
@en
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P1476
Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.
@en
P2093
Garcia-Blanco MA
Mansfield SG
Mitchell LG
Puttaraju M
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P2888
P304
P356
10.1038/SJ.GT.3301307
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2000-11-01T00:00:00Z
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P6179
1026544461