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MECHANISMS IN ENDOCRINOLOGY: Alternative splicing: the new frontier in diabetes researchNew approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicalsAntisense mediated splicing modulation for inherited metabolic diseases: challenges for deliveryAlternative splicing for diseases, cancers, drugs, and databasesAnimal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyLong non-coding RNA INXS is a critical mediator of BCL-XS induced apoptosisRecent advances using zebrafish animal models for muscle disease drug discoveryThe alternative heart: impact of alternative splicing in heart disease.LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblastsDesign and evaluation of locked nucleic acid-based splice-switching oligonucleotides in vitro.Detecting and characterizing circular RNAs.Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia modelSpinal Muscular Atrophy Therapeutics: Where do we Stand?A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver.Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressivaFunctional analysis of deep intronic SNP rs13438494 in intron 24 of PCLO gene.ALS-associated FUS mutations result in compromised FUS alternative splicing and autoregulation.Zinc finger protein 407 (ZFP407) regulates insulin-stimulated glucose uptake and glucose transporter 4 (Glut4) mRNAFunctional characterization of the spf/ash splicing variation in OTC deficiency of mice and man.Alternative RNA splicing and cancerDeciphering targeting rules of splicing modulator compounds: case of TG003.Mcl-1 involvement in mitochondrial dynamics is associated with apoptotic cell deathTranscriptomic analysis of PNN- and ESRP1-regulated alternative pre-mRNA splicing in human corneal epithelial cellsOncogene- and drug resistance-associated alternative exon usage in acute myeloid leukemia (AML).Aryl hydrocarbon receptor nuclear translocator (ARNT) isoforms control lymphoid cancer cell proliferation through differentially regulating tumor suppressor p53 activity.Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy.TGF-β1 Induces Polypyrimidine Tract-Binding Protein to Alter Fibroblasts Proliferation and Fibronectin Deposition in Keloid.Molecular genetics of high-risk chronic lymphocytic leukemia.Ataxin-3 protein and RNA toxicity in spinocerebellar ataxia type 3: current insights and emerging therapeutic strategies.New developments in exon skipping and splice modulation therapies for neuromuscular diseases.Pre-mRNA splicing in cancer: the relevance in oncogenesis, treatment and drug resistance.Antisense oligonucleotide-mediated exon skipping as a strategy to reduce proteolytic cleavage of ataxin-3.Redirecting splicing with bifunctional oligonucleotides.Protein Sam68 regulates the alternative splicing of survivin DEx3.Novel splice-switching oligonucleotide promotes BRCA1 aberrant splicing and susceptibility to PARP inhibitor action.Managing the sequence-specificity of antisense oligonucleotides in drug discovery.Regulation of a strong F9 cryptic 5'ss by intrinsic elements and by combination of tailored U1snRNAs with antisense oligonucleotidesCytokine-induced slowing of STAT3 nuclear import; faster basal trafficking of the STAT3β isoform.Exploring monovalent and multivalent peptides for the inhibition of FBP21-tWW.Nanotherapy for Duchenne muscular dystrophy.
P2860
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P2860
description
article científic
@ca
article scientifique
@fr
articol științific
@ro
articolo scientifico
@it
artigo científico
@gl
artigo científico
@pt
artigo científico
@pt-br
artikel ilmiah
@id
artikull shkencor
@sq
artículo científico
@es
name
Splice modulating therapies for human disease.
@en
Splice modulating therapies for human disease.
@nl
type
label
Splice modulating therapies for human disease.
@en
Splice modulating therapies for human disease.
@nl
prefLabel
Splice modulating therapies for human disease.
@en
Splice modulating therapies for human disease.
@nl
P1433
P1476
Splice modulating therapies for human disease.
@en
P304
P356
10.1016/J.CELL.2012.02.014
P407
P577
2012-03-01T00:00:00Z