Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides.
about
Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patientsAntisense-mediated exon skipping: a versatile tool with therapeutic and research applicationsFunctional studies on the ATM intronic splicing processing elementNew approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicalsRNA and diseaseSplice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia modelRestoration of the cystic fibrosis transmembrane conductance regulator function by splicing modulation.Anti-miRNA oligonucleotides (AMOs): ammunition to target miRNAs implicated in human disease?Inhibition of nonsense-mediated mRNA decay by antisense morpholino oligonucleotides restores functional expression of hERG nonsense and frameshift mutations in long-QT syndromeAlternative splicing impairs soluble guanylyl cyclase function in aortic aneurysmThe potential of antisense as a CNS therapeutic.Macro and small over micro: macromolecules and small molecules that regulate microRNAs.Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing.Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides.AON-mediated Exon Skipping Restores Ciliation in Fibroblasts Harboring the Common Leber Congenital Amaurosis CEP290 Mutation.Propionic and methylmalonic acidemia: antisense therapeutics for intronic variations causing aberrantly spliced messenger RNATargeting RNA splicing for disease therapy.Nuclear antisense effects in cyclophilin A pre-mRNA splicing by oligonucleotides: a comparison of tricyclo-DNA with LNAAlternative splicing and disease.Neuronal ceroid lipofuscinosis type CLN2: a new rationale for the construction of phenotypic subgroups based on a survey of 25 cases in South America.Alternative splicing: role of pseudoexons in human disease and potential therapeutic strategies.New insights in gene-derived therapy: the example of Duchenne muscular dystrophy.RNA splicing: disease and therapy.Cystic fibrosis genetics: from molecular understanding to clinical application.Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing.Correction of alternative splicing of tau in frontotemporal dementia and parkinsonism linked to chromosome 17.In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs.Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment.Regulation of telomerase alternative splicing: a target for chemotherapyDeep intronic mutations and human disease.Temperature-dependent splicing of beta-globin pre-mRNA.Antisense therapeutics for neurofibromatosis type 1 caused by deep intronic mutations.hnRNP H binding at the 5' splice site correlates with the pathological effect of two intronic mutations in the NF-1 and TSHbeta genesRepair of CFTR mRNA by spliceosome-mediated RNA trans-splicing.Cas9/gRNA targeted excision of cystic fibrosis-causing deep-intronic splicing mutations restores normal splicing of CFTR mRNA.In vitro antisense therapeutics for a deep intronic mutation causing Neurofibromatosis type 2.Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells.Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency.Hybrid splicing minigene and antisense oligonucleotides as efficient tools to determine functional protein/RNA interactions.
P2860
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P2860
Correction of aberrant splicing of the cystic fibrosis transmembrane conductance regulator (CFTR) gene by antisense oligonucleotides.
description
1999 nî lūn-bûn
@nan
1999年の論文
@ja
1999年論文
@yue
1999年論文
@zh-hant
1999年論文
@zh-hk
1999年論文
@zh-mo
1999年論文
@zh-tw
1999年论文
@wuu
1999年论文
@zh
1999年论文
@zh-cn
name
Correction of aberrant splicin ...... by antisense oligonucleotides.
@en
Correction of aberrant splicin ...... membrane conductance regulator
@nl
type
label
Correction of aberrant splicin ...... by antisense oligonucleotides.
@en
Correction of aberrant splicin ...... membrane conductance regulator
@nl
prefLabel
Correction of aberrant splicin ...... by antisense oligonucleotides.
@en
Correction of aberrant splicin ...... membrane conductance regulator
@nl
P2093
P2860
P356
P1476
Correction of aberrant splicin ...... by antisense oligonucleotides.
@en
P2093
P2860
P304
36193-36199
P356
10.1074/JBC.274.51.36193
P407
P577
1999-12-01T00:00:00Z