Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
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Title
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
Authors
Keywords
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Journal
GENE THERAPY
Volume -, Issue -, Pages -
Publisher
Springer Science and Business Media LLC
Online
2022-01-25
DOI
10.1038/s41434-022-00316-7
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Note: Only part of the references are listed.- Transcriptome alterations in myotonic dystrophy frontal cortex
- (2021) Brittney A. Otero et al. Cell Reports
- Deciphering the mechanisms underlying brain alterations and cognitive impairment in congenital myotonic dystrophy
- (2021) Thiéry De Serres-Bérard et al. NEUROBIOLOGY OF DISEASE
- Systemic therapy in an RNA toxicity mouse model with an antisense oligonucleotide therapy targeting a non-CUG sequence within the DMPK 3′UTR RNA
- (2020) Ramesh S Yadava et al. HUMAN MOLECULAR GENETICS
- Deprivation of Muscleblind-Like Proteins Causes Deficits in Cortical Neuron Distribution and Morphological Changes in Dendritic Spines and Postsynaptic Densities
- (2019) Kuang-Yung Lee et al. Frontiers in Neuroanatomy
- Differentiation of lymphoblastoid-derived iPSCs into functional cardiomyocytes, neurons and myoblasts
- (2019) Hugo Poulin et al. BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
- Cell-type-specific dysregulation of RNA alternative splicing in short tandem repeat mouse knockin models of myotonic dystrophy
- (2019) Curtis A. Nutter et al. GENES & DEVELOPMENT
- Dosage effect of multiple genes accounts for multisystem disorder of myotonic dystrophy type 1
- (2019) Qi Yin et al. CELL RESEARCH
- Lymphoblastoids cell lines – Derived iPSC line from a 26-year-old myotonic dystrophy type 1 patient carrying (CTG) 200 expansion in the DMPK gene: CHUQi001-A
- (2018) Laurie Martineau et al. Stem Cell Research
- Generation of Neural Cells from DM1 Induced Pluripotent Stem Cells As Cellular Model for the Study of Central Nervous System Neuropathogenesis
- (2018) Guangbin Xia et al. Cellular Reprogramming
- Spinal muscular atrophy approval boosts antisense drugs
- (2017) Elie Dolgin NATURE BIOTECHNOLOGY
- Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice
- (2017) Dominic Jauvin et al. Molecular Therapy-Nucleic Acids
- Congenital myotonic dystrophy: ventriculomegaly and shunt considerations for the pediatric neurosurgeon
- (2016) Ian S. Mutchnick et al. CHILDS NERVOUS SYSTEM
- Dmpkgene deletion or antisense knockdown does not compromise cardiac or skeletal muscle function in mice
- (2016) Samuel T. Carrell et al. HUMAN MOLECULAR GENETICS
- Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy
- (2016) Yuanzheng Gao et al. MOLECULAR THERAPY
- Disease burden and functional outcomes in congenital myotonic dystrophy
- (2016) Nicholas E. Johnson et al. NEUROLOGY
- Therapeutic Approaches for Dominant Muscle Diseases: Highlight on Myotonic Dystrophy
- (2015) A. Klein et al. CURRENT GENE THERAPY
- Dynamic changes of nuclear RNA foci in proliferating DM1 cells
- (2015) Guangbin Xia et al. HISTOCHEMISTRY AND CELL BIOLOGY
- Abnormal sodium current properties contribute to cardiac electrical and contractile dysfunction in a mouse model of myotonic dystrophy type 1
- (2015) Vincent Algalarrondo et al. NEUROMUSCULAR DISORDERS
- Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy Type 1 Induced Pluripotent Stem Cell-Derived Neural Stem Cells
- (2015) Guangbin Xia et al. STEM CELLS
- MBNL Sequestration by Toxic RNAs and RNA Misprocessing in the Myotonic Dystrophy Brain
- (2015) Marianne Goodwin et al. Cell Reports
- Loss of MBNL Leads to Disruption of Developmentally Regulated Alternative Polyadenylation in RNA-Mediated Disease
- (2014) Ranjan Batra et al. MOLECULAR CELL
- Prevalence of Muscular Dystrophies: A Systematic Literature Review
- (2014) Alice Theadom et al. NEUROEPIDEMIOLOGY
- RNA toxicity in human disease and animal models: From the uncovering of a new mechanism to the development of promising therapies
- (2013) Géraldine Sicot et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE
- Myotonic dystrophy CTG expansion affects synaptic vesicle proteins, neurotransmission and mouse behaviour
- (2013) Oscar Hernández-Hernández et al. BRAIN
- Compound loss of muscleblind-like function in myotonic dystrophy
- (2013) Kuang-Yung Lee et al. EMBO Molecular Medicine
- The Impact of Congenital and Childhood Myotonic Dystrophy on Quality of Life
- (2013) Nicholas E. Johnson et al. JOURNAL OF CHILD NEUROLOGY
- Age of Onset of RNA Toxicity Influences Phenotypic Severity: Evidence from an Inducible Mouse Model of Myotonic Dystrophy (DM1)
- (2013) Jordan T. Gladman et al. PLoS One
- Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals
- (2013) Gene Hung et al. Nucleic Acid Therapeutics
- Transcriptome-wide Regulation of Pre-mRNA Splicing and mRNA Localization by Muscleblind Proteins
- (2012) Eric T. Wang et al. CELL
- Targeting nuclear RNA for in vivo correction of myotonic dystrophy
- (2012) Thurman M. Wheeler et al. NATURE
- Muscleblind-like 2-Mediated Alternative Splicing in the Developing Brain and Dysregulation in Myotonic Dystrophy
- (2012) Konstantinos Charizanis et al. NEURON
- Molecular, Physiological, and Motor Performance Defects in DMSXL Mice Carrying >1,000 CTG Repeats from the Human DM1 Locus
- (2012) Aline Huguet et al. PLoS Genetics
- Zebrafish deficient for Muscleblind-like 2 exhibit features of myotonic dystrophy
- (2011) L. E. Machuca-Tzili et al. Disease Models & Mechanisms
- Myotonic dystrophy, when simple repeats reveal complex pathogenic entities: new findings and future challenges
- (2011) G. Sicot et al. HUMAN MOLECULAR GENETICS
- Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
- (2010) Y. Hua et al. GENES & DEVELOPMENT
- Biophysical Properties of Human Nav1.7 Splice Variants and Their Regulation by Protein Kinase A
- (2008) Aurélien Chatelier et al. JOURNAL OF NEUROPHYSIOLOGY
- Electrocardiographic Abnormalities and Sudden Death in Myotonic Dystrophy Type 1
- (2008) William J. Groh et al. NEW ENGLAND JOURNAL OF MEDICINE
- Genetic and Chemical Modifiers of a CUG Toxicity Model in Drosophila
- (2008) Amparo Garcia-Lopez et al. PLoS One
- Expanded CTG repeats within the DMPK 3' UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy
- (2008) J. P. Orengo et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- A postnatal switch of CELF and MBNL proteins reprograms alternative splicing in the developing heart
- (2008) A. Kalsotra et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
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