Pages that link to "Q42218490"
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The following pages link to Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models (Q42218490):
Displaying 33 items.
- Spinal Muscular Atrophy (Q28080774) (← 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)
- Therapeutic Potential of Tricyclo-DNA antisense oligonucleotides (Q28818017) (← links)
- Report on the 3rd Ottawa International Conference on Neuromuscular Biology, Disease and Therapy - September 24-26, 2015, Ottawa, Canada. (Q30355125) (← links)
- Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy. (Q30743508) (← links)
- Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy (Q30821687) (← links)
- Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment (Q33635326) (← links)
- Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy (Q33786251) (← links)
- Spinal Muscular Atrophy Therapeutics: Where do we Stand? (Q34460251) (← links)
- Targeting SR proteins improves SMN expression in spinal muscular atrophy cells (Q34704344) (← links)
- Splicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions (Q36297097) (← links)
- Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy (Q36322222) (← links)
- Splice-switching antisense oligonucleotides as therapeutic drugs (Q37211004) (← links)
- ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice (Q37417518) (← links)
- Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy (Q37677406) (← links)
- The role of antisense oligonucleotide therapy in patients with familial hypercholesterolemia: risks, benefits, and management recommendations (Q38268342) (← links)
- Spinal muscular atrophy--recent therapeutic advances for an old challenge. (Q38489298) (← links)
- Developing therapies for spinal muscular atrophy (Q38546908) (← links)
- Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease. (Q38734734) (← links)
- Pharmacology of Modulators of Alternative Splicing (Q38776406) (← links)
- Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy (Q38843108) (← links)
- Advances in modeling and treating spinal muscular atrophy (Q38912516) (← links)
- Translational development of splice-modifying antisense oligomers (Q38996657) (← links)
- How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy (Q39293468) (← links)
- Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases (Q39391538) (← links)
- Modulation of nuclear REST by alternative splicing: a potential therapeutic target for Huntington's disease (Q44573861) (← links)
- Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis (Q47152662) (← links)
- Oligonucleotide therapies for disorders of the nervous system (Q48288187) (← links)
- SMN deficiency negatively impacts red pulp macrophages and spleen development in mouse models of spinal muscular atrophy (Q50219618) (← links)
- Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript. (Q55266977) (← links)
- Blocking p62-dependent SMN degradation ameliorates spinal muscular atrophy disease phenotypes (Q57245726) (← links)
- Drug treatment for spinal muscular atrophy types II and III (Q89477851) (← links)
- Functional characterization of SMN evolution in mouse models of SMA (Q91529446) (← links)