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MicroRNA-Based Therapeutic Perspectives in Myotonic Dystrophy

International journal of molecular sciences, 2019-11, Vol.20 (22), p.5600

2019

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Deciphering the Complex Molecular Pathogenesis of Myotonic Dystrophy Type 1 through Omics Studies

International journal of molecular sciences, 2022-01, Vol.23 (3), p.1441

2022

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Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSALR Mice

International journal of molecular sciences, 2023-06, Vol.24 (12), p.9820

2023

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Drosophila SMN2 minigene reporter model identifies moxifloxacin as a candidate therapy for SMA

The FASEB journal, 2020-02, Vol.34 (2), p.3021-3036

2020

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BlockmiR AONs as Site-Specific Therapeutic MBNL Modulation in Myotonic Dystrophy 2D and 3D Muscle Cells and HSALR Mice

Pharmaceutics, 2023-03, Vol.15 (4), p.1118

2023

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Rabphilin silencing causes dilated cardiomyopathy in a Drosophila model of nephrocyte damage

Scientific reports, 2021-07, Vol.11 (1), p.15287-15287, Article 15287

2021

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The hallmarks of myotonic dystrophy type 1 muscle dysfunction

Biological reviews of the Cambridge Philosophical Society, 2021-04, Vol.96 (2), p.716-730

2021

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Gender as a Modifying Factor Influencing Myotonic Dystrophy Type 1 Phenotype Severity and Mortality: A Nationwide Multiple Databases Cross-Sectional Observational Study

PloS one, 2016-02, Vol.11 (2), p.e0148264-e0148264

2016

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Plasmatic extracellular vesicle microRNAs in malignant pleural mesothelioma and asbestos-exposed subjects suggest a 2-miRNA signature as potential biomarker of disease

PloS one, 2017-05, Vol.12 (5), p.e0176680

2017

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Gene Expression Profiling in Slow-Type Calf Soleus Muscle of 30 Days Space-Flown Mice

PloS one, 2017-01, Vol.12 (1), p.e0169314-e0169314

2017

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Renal Lipotoxicity-Associated Inflammation and Insulin Resistance Affects Actin Cytoskeleton Organization in Podocytes

PloS one, 2015-11, Vol.10 (11), p.e0142291

2015

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Characterisation of CDKL5 Transcript Isoforms in Human and Mouse

PloS one, 2016-06, Vol.11 (6), p.e0157758-e0157758

2016

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Genetic and chemical modifiers of a CUG toxicity model in Drosophila

PloS one, 2008-02, Vol.3 (2), p.e1595

2008

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Rabphilin involvement in filtration and molecular uptake in Drosophila nephrocytes suggests a similar role in human podocytes

Disease models & mechanisms, 2020-09, Vol.13 (9)

2020

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Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy

Molecular therapy. Nucleic acids, 2020-09, Vol.21, p.837-849

2020

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RNA-mediated therapies in myotonic dystrophy

Drug discovery today, 2018-12, Vol.23 (12), p.2013-2022

2018

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"I Know that You Know that I Know": Neural Substrates Associated with Social Cognition Deficits in DM1 Patients

PloS one, 2016-06, Vol.11 (6), p.e0156901-e0156901

2016

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Patients experiencing statin-induced myalgia exhibit a unique program of skeletal muscle gene expression following statin re-challenge

PloS one, 2017-08, Vol.12 (8), p.e0181308-e0181308

2017

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miR-23b and miR-218 silencing increase Muscleblind-like expression and alleviate myotonic dystrophy phenotypes in mammalian models

Nature communications, 2018-06, Vol.9 (1), p.2482-13, Article 2482

2018

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Expanded CCUG repeat RNA expression in Drosophila heart and muscle trigger Myotonic Dystrophy type 1-like phenotypes and activate autophagocytosis genes

Scientific reports, 2017-06, Vol.7 (1), p.2843-13, Article 2843

2017

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Myotonic dystrophy: candidate small molecule therapeutics

Drug discovery today, 2017-11, Vol.22 (11), p.1740-1748

2017

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Differences in splicing defects between the grey and white matter in myotonic dystrophy type 1 patients

PloS one, 2020-05, Vol.15 (5), p.e0224912-e0224912

2020

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Mutation analysis of multiple pilomatricomas in a patient with myotonic dystrophy type 1 suggests a DM1-associated hypermutation phenotype

PloS one, 2020-03, Vol.15 (3), p.e0230003-e0230003

2020

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Learning epistatic gene interactions from perturbation screens

PloS one, 2021-07, Vol.16 (7), p.e0254491-e0254491

2021

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Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSA[sup.LR] Mice

International journal of molecular sciences, 2023-06, Vol.24 (12)

2023

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MicroRNA-Based Therapeutic Perspectives in Myotonic Dystrophy

Deciphering the Complex Molecular Pathogenesis of Myotonic Dystrophy Type 1 through Omics Studies

Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSALR Mice

Drosophila SMN2 minigene reporter model identifies moxifloxacin as a candidate therapy for SMA

BlockmiR AONs as Site-Specific Therapeutic MBNL Modulation in Myotonic Dystrophy 2D and 3D Muscle Cells and HSALR Mice

Rabphilin silencing causes dilated cardiomyopathy in a Drosophila model of nephrocyte damage

The hallmarks of myotonic dystrophy type 1 muscle dysfunction

Gender as a Modifying Factor Influencing Myotonic Dystrophy Type 1 Phenotype Severity and Mortality: A Nationwide Multiple Databases Cross-Sectional Observational Study

Plasmatic extracellular vesicle microRNAs in malignant pleural mesothelioma and asbestos-exposed subjects suggest a 2-miRNA signature as potential biomarker of disease

Gene Expression Profiling in Slow-Type Calf Soleus Muscle of 30 Days Space-Flown Mice

Renal Lipotoxicity-Associated Inflammation and Insulin Resistance Affects Actin Cytoskeleton Organization in Podocytes

Characterisation of CDKL5 Transcript Isoforms in Human and Mouse

Genetic and chemical modifiers of a CUG toxicity model in Drosophila

Rabphilin involvement in filtration and molecular uptake in Drosophila nephrocytes suggests a similar role in human podocytes

Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy

RNA-mediated therapies in myotonic dystrophy

"I Know that You Know that I Know": Neural Substrates Associated with Social Cognition Deficits in DM1 Patients

Patients experiencing statin-induced myalgia exhibit a unique program of skeletal muscle gene expression following statin re-challenge

miR-23b and miR-218 silencing increase Muscleblind-like expression and alleviate myotonic dystrophy phenotypes in mammalian models

Expanded CCUG repeat RNA expression in Drosophila heart and muscle trigger Myotonic Dystrophy type 1-like phenotypes and activate autophagocytosis genes

Myotonic dystrophy: candidate small molecule therapeutics

Differences in splicing defects between the grey and white matter in myotonic dystrophy type 1 patients

Mutation analysis of multiple pilomatricomas in a patient with myotonic dystrophy type 1 suggests a DM1-associated hypermutation phenotype

Learning epistatic gene interactions from perturbation screens

Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSA[sup.LR] Mice

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