Pages that link to "Q34168139"
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The following pages link to Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy (Q34168139):
Displaying 50 items.
- Facioscapulohumeral dystrophy: the path to consensus on pathophysiology (Q21195884) (← links)
- Spinal muscular atrophy (Q21202863) (← links)
- The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy (Q24604444) (← links)
- Neuromuscular Junctions as Key Contributors and Therapeutic Targets in Spinal Muscular Atrophy (Q26766502) (← links)
- Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the Brain (Q26770355) (← links)
- Spinal muscular atrophy: from gene discovery to clinical trials (Q26824894) (← links)
- Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery (Q26830637) (← links)
- Involvement of long noncoding RNAs in diseases affecting the central nervous system (Q26852160) (← links)
- Spinal muscular atrophy: development and implementation of potential treatments (Q26861377) (← links)
- A chemical view of oligonucleotides for exon skipping and related drug applications (Q26863684) (← links)
- Applicability of histone deacetylase inhibition for the treatment of spinal muscular atrophy (Q26864605) (← links)
- Antisense oligonucleotides: treating neurodegeneration at the level of RNA (Q27007056) (← links)
- Molecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA) (Q27007782) (← links)
- Splicing therapy for neuromuscular disease (Q27009581) (← links)
- Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides (Q27314897) (← links)
- A large animal model of spinal muscular atrophy and correction of phenotype (Q27340106) (← links)
- Applications of Cas9 as an RNA-programmed RNA-binding protein (Q28083457) (← links)
- Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study (Q28141072) (← links)
- Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response (Q28266165) (← links)
- Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients (Q28542911) (← links)
- Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides (Q28551647) (← links)
- Collaboration for rare disease drug discovery research (Q28650560) (← links)
- Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study (Q28730280) (← links)
- Candidate proteins, metabolites and transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) clinical study (Q28730283) (← links)
- Targeting several CAG expansion diseases by a single antisense oligonucleotide (Q28740870) (← links)
- RNA therapeutics: RNAi and antisense mechanisms and clinical applications (Q28829240) (← links)
- Overcoming cellular barriers for RNA therapeutics (Q30235525) (← links)
- Giant axonal neuropathy: An updated perspective on its pathology and pathogenesis. (Q30363784) (← links)
- Sodium vanadate combined with L-ascorbic acid delays disease progression, enhances motor performance, and ameliorates muscle atrophy and weakness in mice with spinal muscular atrophy. (Q30540653) (← links)
- The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models (Q30546337) (← links)
- Pathological impact of SMN2 mis-splicing in adult SMA mice (Q30551534) (← links)
- Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy. (Q30743508) (← links)
- Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models (Q33635375) (← links)
- Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates (Q33750824) (← links)
- LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts (Q33807788) (← links)
- Defining the therapeutic window in a severe animal model of spinal muscular atrophy (Q33991731) (← links)
- Targeting Splicing in the Treatment of Myelodysplastic Syndromes and Other Myeloid Neoplasms. (Q34047259) (← links)
- Assays for the identification and prioritization of drug candidates for spinal muscular atrophy (Q34082155) (← links)
- Structure/affinity studies in the bicyclo-DNA series: Synthesis and properties of oligonucleotides containing bc(en)-T and iso-tricyclo-T nucleosides (Q34082208) (← links)
- Temporal requirement for high SMN expression in SMA mice (Q34192528) (← links)
- Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders (Q34205620) (← links)
- New therapeutic approaches to spinal muscular atrophy (Q34237525) (← links)
- A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse (Q34241874) (← links)
- RNA therapeutics: beyond RNA interference and antisense oligonucleotides (Q34248493) (← links)
- Therapeutic strategies for the treatment of spinal muscular atrophy. (Q34296140) (← links)
- SMN-inducing compounds for the treatment of spinal muscular atrophy (Q34312156) (← links)
- Antisense oligonucleotides for the treatment of spinal muscular atrophy (Q34336279) (← links)
- Limited phenotypic effects of selectively augmenting the SMN protein in the neurons of a mouse model of severe spinal muscular atrophy (Q34431129) (← links)
- Mechanisms and Regulation of Alternative Pre-mRNA Splicing (Q34467642) (← links)
- A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes (Q34490238) (← links)