A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.
about
Applicability of histone deacetylase inhibition for the treatment of spinal muscular atrophySpinal Muscular AtrophySMN in spinal muscular atrophy and snRNP biogenesisTrans-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.Spinal muscular atrophy: mechanisms and therapeutic strategies.Splicing of the Survival Motor Neuron genes and implications for treatment of SMATherapeutic developments in spinal muscular atrophy.Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development.Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice.SMN-inducing compounds for the treatment of spinal muscular atrophy.Antisense oligonucleotides for the treatment of spinal muscular atrophy.Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.Absence of an intron splicing silencer in porcine Smn1 intron 7 confers immunity to the exon skipping mutation in human SMN2.Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophyMechanistic principles of antisense targets for the treatment of spinal muscular atrophyRecent developments in the treatment of Duchenne muscular dystrophy and spinal muscular atrophy.Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy.Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model.Spinal muscular atrophy: a timely review.Splicing therapeutics in SMN2 and APOB.Targeting RNA-splicing for SMA treatment.Progress in therapeutic antisense applications for neuromuscular disorders.Dual masking of specific negative splicing regulatory elements resulted in maximal exon 7 inclusion of SMN2 gene.Opportunities and challenges for the development of antisense treatment in neuromuscular disorders.Targeting RNA to treat neuromuscular disease.Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain.Mouse models of SMA: tools for disease characterization and therapeutic development.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Redirecting splicing with bifunctional oligonucleotides.Investigational therapies for the treatment of spinal muscular atrophy.Developing therapies for spinal muscular atrophy.Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.Blocking p62-dependent SMN degradation ameliorates spinal muscular atrophy disease phenotypes
P2860
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P2860
A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.
description
2008 nî lūn-bûn
@nan
2008 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած
@hyw
2008 թվականի նոյեմբերին հրատարակված գիտական հոդված
@hy
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
name
A negatively acting bifunction ...... ron both in vitro and in vivo.
@ast
A negatively acting bifunction ...... ron both in vitro and in vivo.
@en
A negatively acting bifunction ...... ron both in vitro and in vivo.
@nl
type
label
A negatively acting bifunction ...... ron both in vitro and in vivo.
@ast
A negatively acting bifunction ...... ron both in vitro and in vivo.
@en
A negatively acting bifunction ...... ron both in vitro and in vivo.
@nl
prefLabel
A negatively acting bifunction ...... ron both in vitro and in vivo.
@ast
A negatively acting bifunction ...... ron both in vitro and in vivo.
@en
A negatively acting bifunction ...... ron both in vitro and in vivo.
@nl
P2093
P2860
P356
P1433
P1476
A negatively acting bifunction ...... ron both in vitro and in vivo.
@en
P2093
Alexa Dickson
Christian L Lorson
Erkan Osman
P2860
P304
P356
10.1089/HUM.2008.067
P577
2008-11-01T00:00:00Z