Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy. - Wikidata - awgldk - TriplyDB

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.

http://www.wikidata.org/entity/Q34540158

about

Emerging therapies and challenges in spinal muscular atrophy Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment GAA Deficiency in Pompe Disease Is Alleviated by Exon Inclusion in iPSC-Derived Skeletal Muscle Cells.Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Using Morpholinos to Control Gene Expression.Viral Vector-Mediated Antisense Therapy for Genetic Diseases.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Myotonic dystrophy: approach to therapy.Translational development of splice-modifying antisense oligomers.How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy The SMA Trust: the role of a disease-focused research charity in developing treatments for SMA.Splice-Switching Therapy for Spinal Muscular Atrophy.Synthetic Nucleic Acid Analogues in Gene Therapy: An Update for Peptide-Oligonucleotide Conjugates.Therapeutic strategies for spinal muscular atrophy: SMN and beyond.Gene suppression approaches to neurodegeneration.Therapies targeting DNA and RNA in Huntington's disease.Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis.Antisense oligonucleotides: the next frontier for treatment of neurological disorders.Making sense of antisense oligonucleotides: A narrative review.Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape.Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy.Oligonucleotide therapies for disorders of the nervous system.Advances in spinal muscular atrophy therapeutics.Interventions Targeting Glucocorticoid-Krüppel-like Factor 15-Branched-Chain Amino Acid Signaling Improve Disease Phenotypes in Spinal Muscular Atrophy Mice.Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics.Light modulation ameliorates expression of circadian genes and disease progression in spinal muscular atrophy mice

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P2860

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.

http://www.wikidata.org/entity/Q34540158

description

2016 nî lūn-bûn

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2016 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած

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2016 թվականի սեպտեմբերին հրատարակված գիտական հոդված

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2016年の論文

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2016年論文

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2016年論文

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2016年論文

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2016年論文

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2016年論文

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2016年论文

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name

Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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type

label

Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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prefLabel

Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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PNAS.1605731113

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Proceedings of the National Academy of Sciences of the United States of America

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Systemic peptide-mediated olig ...... al in spinal muscular atrophy.

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Amer F Saleh

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Fazel Shabanpoor

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Francesco Muntoni

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Gareth Hazell

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Haiyan Zhou

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Katharina E Meijboom

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Michael J Gait

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P2860

The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins

The SMN-SIP1 complex has an essential role in spliceosomal snRNP biogenesis

Genetic therapies for RNA mis-splicing diseases

An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients

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

SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy

Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients

Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides

Alpha-synuclein protects cerebellar granule neurons against 6-hydroxydopamine-induced death

Identification and characterization of a spinal muscular atrophy-determining gene

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10962-10967

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39

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James N. Sleigh

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Suzan M Hammond

Matthew J. Wood

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2016-09-12T00:00:00Z

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27621445

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spinal muscular atrophy

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5047168

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