Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response - Wikidata - awgldk - TriplyDB

Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response

Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response

http://www.wikidata.org/entity/Q28266165

about

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment.The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy Survival Motor Neuron (SMN) protein is required for normal mouse liver development.Alternatively spliced mu opioid receptor C termini impact the diverse actions of morphine.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Advances in modeling and treating spinal muscular atrophy How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy Splice-Switching Therapy for Spinal Muscular Atrophy.Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset.Therapeutic strategies for spinal muscular atrophy: SMN and beyond.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Time Is Motor Neuron: Therapeutic Window and Its Correlation with Pathogenetic Mechanisms in Spinal Muscular Atrophy.Making sense of antisense oligonucleotides: A narrative review.Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy.Intracerebroventricular Delivery in Mice for Motor Neuron Diseases.Antisense oligonucleotides in neurological disorders.

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Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response

http://www.wikidata.org/entity/Q28266165

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2015 nî lūn-bûn

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2015 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած

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2015 թվականի նոյեմբերին հրատարակված գիտական հոդված

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2015年の論文

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2015年論文

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2015年論文

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2015年論文

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Repeated low doses of morpholi ...... window of therapeutic response

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Repeated low doses of morpholi ...... window of therapeutic response

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Repeated low doses of morpholi ...... window of therapeutic response

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Repeated low doses of morpholi ...... window of therapeutic response

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Repeated low doses of morpholi ...... window of therapeutic response

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Repeated low doses of morpholi ...... window of therapeutic response

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Repeated low doses of morpholi ...... window of therapeutic response

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Repeated low doses of morpholi ...... window of therapeutic response

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Human Molecular Genetics

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Repeated low doses of morpholi ...... window of therapeutic response

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Elena Marrosu

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Haiyan Zhou

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Jennifer Morgan

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Jinhong Meng

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Narinder Janghra

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P2860

Survival motor neuron gene 2 silencing by DNA methylation correlates with spinal muscular atrophy disease severity and can be bypassed by histone deacetylase inhibition

Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy

Synaptic defects in the spinal and neuromuscular circuitry in a mouse model of spinal muscular atrophy

A large animal model of spinal muscular atrophy and correction of phenotype.

LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy.

Identification and characterization of a spinal muscular atrophy-determining gene

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

CNS-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 mice

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scholarly article

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10.1093/HMG/DDV329

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22

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24

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Francesco Muntoni

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2015-11-15T00:00:00Z

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26264577

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spinal muscular atrophy

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4614699

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