Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.
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Four decades of neurodegenerative disease research: how far we have come!Synthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseasesSpinal muscular atrophy: development and implementation of potential treatmentsA chemical view of oligonucleotides for exon skipping and related drug applicationsAntisense oligonucleotides: treating neurodegeneration at the level of RNAMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Splicing therapy for neuromuscular diseaseSpinal Muscular AtrophyProlactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway.SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophyUtility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analysesSMN in spinal muscular atrophy and snRNP biogenesisPostsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.Guanine analogues enhance antisense oligonucleotide-induced exon skipping in dystrophin gene in vitro and in vivo.Trans-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: new and emerging insights from model miceAn antisense microwalk reveals critical role of an intronic position linked to a unique long-distance interaction in pre-mRNA splicingSpinal muscular atrophy: mechanisms and therapeutic strategies.Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse modelSplicing of the Survival Motor Neuron genes and implications for treatment of SMAAntisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe 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.A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouseRNA therapeutics: beyond RNA interference and antisense oligonucleotides.SMN-inducing compounds for the treatment of spinal muscular atrophy.Potent and selective inhibition of A-to-I RNA editing with 2'-O-methyl/locked nucleic acid-containing antisense oligoribonucleotides.Antisense oligonucleotides for the treatment of spinal muscular atrophy.Spinal Muscular Atrophy Therapeutics: Where do we Stand?A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes.Antisense-mediated isoform switching of steroid receptor coactivator-1 in the central nucleus of the amygdala of the mouse brain.ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Early functional impairment of sensory-motor connectivity in a mouse model of spinal muscular atrophy.Efficient in vivo manipulation of alternative pre-mRNA splicing events using antisense morpholinos in mice.Regulation of the mutually exclusive exons 8a and 8 in the CaV1.2 calcium channel transcript by polypyrimidine tract-binding protein.Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compoundsRepair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.Spinal muscular atrophy: from tissue specificity to therapeutic strategies
P2860
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P2860
Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy.
description
2009 nî lūn-bûn
@nan
2009年の論文
@ja
2009年学术文章
@wuu
2009年学术文章
@zh
2009年学术文章
@zh-cn
2009年学术文章
@zh-hans
2009年学术文章
@zh-my
2009年学术文章
@zh-sg
2009年學術文章
@yue
2009年學術文章
@zh-hant
name
Oligonucleotide-mediated survi ...... el of spinal muscular atrophy.
@en
Oligonucleotide-mediated survi ...... el of spinal muscular atrophy.
@nl
type
label
Oligonucleotide-mediated survi ...... el of spinal muscular atrophy.
@en
Oligonucleotide-mediated survi ...... el of spinal muscular atrophy.
@nl
prefLabel
Oligonucleotide-mediated survi ...... el of spinal muscular atrophy.
@en
Oligonucleotide-mediated survi ...... el of spinal muscular atrophy.
@nl
P2093
P1476
Oligonucleotide-mediated survi ...... del of spinal muscular atrophy
@en
P2093
Carlyn A Patterson
Gordon J Lutz
Jason H Williams
Melanie K Tallent
Rebecca C Schray
Semira O Ayitey
P304
P356
10.1523/JNEUROSCI.0950-09.2009
P407
P577
2009-06-01T00:00:00Z