Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
about
Development of a single vector system that enhances trans-splicing of SMN2 transcriptsDrug treatment for spinal muscular atrophy types II and IIIDrug treatment for spinal muscular atrophy types II and IIIThe splicing regulator Sam68 binds to a novel exonic splicing silencer and functions in SMN2 alternative splicing in spinal muscular atrophyA positive modifier of spinal muscular atrophy in the SMN2 geneThe RNA binding protein hnRNP Q modulates the utilization of exon 7 in the survival motor neuron 2 (SMN2) geneDifferential 3' splice site recognition of SMN1 and SMN2 transcripts by U2AF and U2 snRNPAntisense-mediated exon skipping: a versatile tool with therapeutic and research applicationsMolecular Mechanisms of Neurodegeneration in Spinal Muscular AtrophyApplicability of histone deacetylase inhibition for the treatment of spinal muscular atrophyMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Splicing therapy for neuromuscular diseaseProgress toward therapy with antisense-mediated splicing modulationIsolated pseudo-RNA-recognition motifs of SR proteins can regulate splicing using a noncanonical mode of RNA recognitionA short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophyUtility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analysesSMN in spinal muscular atrophy and snRNP biogenesisCNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.Pathological impact of SMN2 mis-splicing in adult SMA miceAlternative splicing of exon 10 in the tau gene as a target for treatment of tauopathies.Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe DiseaseTrans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblastsSpinal muscular atrophy: mechanisms and therapeutic strategies.A renaissance for antisense oligonucleotide drugs in neurology: exon skipping breaks new groundAntisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse modelThe BioAssay network and its implications to future therapeutic discovery.Splicing of the Survival Motor Neuron genes and implications for treatment of SMAAssays for the identification and prioritization of drug candidates for spinal muscular atrophySplice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia modelA negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.Therapeutic developments in spinal muscular atrophy.Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.Engineering splicing factors with designed specificitiesRNA therapeutics: beyond RNA interference and antisense oligonucleotides.A feedback loop regulates splicing of the spinal muscular atrophy-modifying gene, SMN2.Spinal Muscular Atrophy Therapeutics: Where do we Stand?SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes.
P2860
Q21092173-8E1B6B1F-F19B-4DEA-8A47-3C0255DA085AQ24200270-ECE66683-A228-4B06-9222-ADB74EC1A6D2Q24234414-FC3B456C-FD7E-4843-8B59-05BE4BDA8A86Q24301061-D256AEF3-843C-46EF-9E1A-BEF0CE7554C9Q24646149-E94C53DF-9666-4921-B4FF-D99E9CC9BAD4Q24648050-FCB1C55F-F0F8-4DE2-88E2-74737EC8A500Q24651863-6E7CBA8E-DE66-408F-AE6E-D2A8E069806AQ24682555-D0EF970D-9C51-4B10-BC75-F3EFA17733B4Q26752798-37FB0FCE-94CA-4801-AC1E-9F88E9C86579Q26864605-6C3730B1-EFE6-4139-BA38-0D06E10867F6Q27007782-CB2C4A86-900B-4FDC-A157-D3B0388F0F88Q27009581-E8D246FA-6578-4A64-BCFA-FD7FCBB14225Q27489807-5C91EE2A-3777-46C2-81A2-E512436E1EF3Q27678986-51C584BF-2064-42C5-BA20-A84D5AFB4237Q28244480-E730F66B-EC05-4531-BADD-23839F618673Q28740896-DFFCF2E7-C28A-49F6-BF4C-E1DA40645C1DQ29013847-F8871AEC-08EA-437B-B028-A9412B0B43A7Q30493891-2E8023A8-25C8-4E1C-9EF9-0612E43D6557Q30551534-2AE9E570-5DDC-4789-AE32-91FE1ED82254Q33393896-71E4AA66-985E-4A27-9BEC-558397FD1A64Q33635367-AF5AF40A-002B-45F5-950E-CA4EB0F75A77Q33720730-03519DA5-9B9B-4F6C-9E26-DB6036D8BD81Q33750824-4929FD45-EDE4-4B02-93E8-29E284B8516CQ33807788-1F860A17-25A4-405B-93E2-4941C3A57824Q33871311-A60A8F44-3896-4C7A-81E9-62DD9475ABBCQ33950888-D00F2C25-6439-41B5-894B-DD18007EB019Q34030144-B93A39C1-63FA-40FA-B83E-1D76C7BE89C0Q34046379-038D923A-AA30-4E6A-BFDA-75C57580B395Q34065993-DBAE2AEB-99F5-4E40-A5AA-47921EB35C83Q34082155-B13CA38E-ADF4-4700-8C93-50C303B86E44Q34117470-613E864C-CBAE-4FBB-9EB8-E945A327BE1BQ34132150-4FB1F134-701E-4DFA-B82F-3B16F9068A15Q34156272-16D8043E-6C36-4AC7-B9F1-E433461FF54AQ34168139-6C057955-6E86-4DE1-B50F-8567A88AB1A4Q34235465-53561CE2-A3F2-4D75-8217-7AC1CE5EC147Q34248493-B9ABF146-9559-4B59-B29C-B25870575397Q34341178-17046493-D37C-4B1D-A791-B1E2324291A9Q34460251-86DC63FA-7FF5-44A3-AACE-BE1E68A92824Q34478749-5DFDBCC2-0356-48EA-9C87-482AE37CD3D9Q34490238-D93E88BD-EACA-4841-AF66-BD2A14120674
P2860
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
description
2007 nî lūn-bûn
@nan
2007 թուականի Ապրիլին հրատարակուած գիտական յօդուած
@hyw
2007 թվականի ապրիլին հրատարակված գիտական հոդված
@hy
2007年の論文
@ja
2007年論文
@yue
2007年論文
@zh-hant
2007年論文
@zh-hk
2007年論文
@zh-mo
2007年論文
@zh-tw
2007年论文
@wuu
name
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
@ast
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
@en
type
label
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
@ast
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
@en
prefLabel
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
@ast
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
@en
P2093
P2860
P1433
P1476
Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon.
@en
P2093
Brenda F Baker
C Frank Bennett
Timothy A Vickers
P2860
P356
10.1371/JOURNAL.PBIO.0050073
P407
P577
2007-04-01T00:00:00Z