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Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients

International journal of molecular sciences, 2024-08, Vol.25 (16), p.8763

2024

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Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches

Nature communications, 2017-11, Vol.8 (1), p.1859-8, Article 1859

2017

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Necroptosis mediates myofibre death in dystrophin-deficient mice

Nature communications, 2018-09, Vol.9 (1), p.3655-10, Article 3655

2018

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Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications

Scientific reports, 2022-03, Vol.12 (1), p.3756-3756, Article 3756

2022

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Gene Editing to Tackle Facioscapulohumeral Muscular Dystrophy

Frontiers in genome editing, 2022-07, Vol.4, p.937879

2022

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Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Journal of cachexia, sarcopenia and muscle, 2020-06, Vol.11 (3), p.768-782

2020

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Targeting the Polyadenylation Signal of Pre-mRNA: A New Gene Silencing Approach for Facioscapulohumeral Dystrophy

International journal of molecular sciences, 2018-05, Vol.19 (5), p.1347

2018

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Genetic Evidence That Captured Retroviral Envelope syncytins Contribute to Myoblast Fusion and Muscle Sexual Dimorphism in Mice

PLoS genetics, 2016-09, Vol.12 (9), p.e1006289

2016

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RIPK3‐mediated cell death is involved in DUX4‐mediated toxicity in facioscapulohumeral dystrophy

Journal of cachexia, sarcopenia and muscle, 2021-12, Vol.12 (6), p.2079-2090

2021

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Therapeutic Strategies Targeting DUX4 in FSHD

Journal of clinical medicine, 2020-09, Vol.9 (9), p.2886

2020

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A Deoxyribonucleic Acid Decoy Trapping DUX4 for the Treatment of Facioscapulohumeral Muscular Dystrophy

Molecular therapy. Nucleic acids, 2020-12, Vol.22, p.1191-1199

2020

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Nuclear protein spreading: implication for pathophysiology of neuromuscular diseases

Human molecular genetics, 2014-08, Vol.23 (15), p.4125-4133

2014

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A Maternal Epimutation of GNAS Leads to Albright Osteodystrophy and Parathyroid Hormone Resistance

The journal of clinical endocrinology and metabolism, 2008-03, Vol.93 (3), p.661-665

2008

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Muscle cells of sporadic amyotrophic lateral sclerosis patients secrete neurotoxic vesicles

Journal of cachexia, sarcopenia and muscle, 2022-04, Vol.13 (2), p.1385-1402

2022

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DUX4 Expression in FSHD Muscles: Focus on Its mRNA Regulation

Journal of personalized medicine, 2020-09, Vol.10 (3), p.73

2020

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Correlation between low FAT1 expression and early affected muscle in facioscapulohumeral muscular dystrophy

Annals of neurology, 2015-09, Vol.78 (3), p.387-400

2015

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Gene Editing Targeting the DUX4 Polyadenylation Signal: A Therapy for FSHD?

Journal of personalized medicine, 2020-12, Vol.11 (1), p.7

2020

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Publisher Correction: Necroptosis mediates myofibre death in dystrophin-deficient mice

Nature communications, 2018-10, Vol.9 (1), p.4107-1, Article 4107

2018

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Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.415-421

2020

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Segregation between SMCHD1 mutation, D4Z4 hypomethylation and Facio-Scapulo-Humeral Dystrophy: a case report

BMC medical genetics, 2016-09, Vol.17 (1), p.66-66, Article 66

2016

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One‐hour universal protocol for mouse genotyping

Muscle & nerve, 2020-06, Vol.61 (6), p.801-807

2020

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Rescue of early-stage myelodysplastic syndrome-deriving erythroid precursors by the ectopic expression of a dominant-negative form of FADD

Blood, 2005-05, Vol.105 (10), p.4035-4042

2005

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Antisense targeting of 3' end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach

Human molecular genetics, 2016-04, Vol.25 (8), p.1468-1478

2016

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Human adipose cells express CD4, CXCR4, and CCR5 receptors: a new target cell type for the immunodeficiency virus‐1?

The FASEB journal, 2002-08, Vol.16 (10), p.1254-1256

2002

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Association Analysis Indicates That a Variant GATA-Binding Site in the PIK3CB Promoter Is a Cis-Acting Expression Quantitative Trait Locus for This Gene and Attenuates Insulin Resistance in Obese Children

Diabetes (New York, N.Y.), 2008-02, Vol.57 (2), p.494-502

2008

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Impact of Disease Severity and Disease-Modifying Therapies on Myostatin Levels in SMA Patients

Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches

Necroptosis mediates myofibre death in dystrophin-deficient mice

Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications

Gene Editing to Tackle Facioscapulohumeral Muscular Dystrophy

Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Targeting the Polyadenylation Signal of Pre-mRNA: A New Gene Silencing Approach for Facioscapulohumeral Dystrophy

Genetic Evidence That Captured Retroviral Envelope syncytins Contribute to Myoblast Fusion and Muscle Sexual Dimorphism in Mice

RIPK3‐mediated cell death is involved in DUX4‐mediated toxicity in facioscapulohumeral dystrophy

Therapeutic Strategies Targeting DUX4 in FSHD

A Deoxyribonucleic Acid Decoy Trapping DUX4 for the Treatment of Facioscapulohumeral Muscular Dystrophy

Nuclear protein spreading: implication for pathophysiology of neuromuscular diseases

A Maternal Epimutation of GNAS Leads to Albright Osteodystrophy and Parathyroid Hormone Resistance

Muscle cells of sporadic amyotrophic lateral sclerosis patients secrete neurotoxic vesicles

DUX4 Expression in FSHD Muscles: Focus on Its mRNA Regulation

Correlation between low FAT1 expression and early affected muscle in facioscapulohumeral muscular dystrophy

Gene Editing Targeting the DUX4 Polyadenylation Signal: A Therapy for FSHD?

Publisher Correction: Necroptosis mediates myofibre death in dystrophin-deficient mice

Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy

Segregation between SMCHD1 mutation, D4Z4 hypomethylation and Facio-Scapulo-Humeral Dystrophy: a case report

One‐hour universal protocol for mouse genotyping

Rescue of early-stage myelodysplastic syndrome-deriving erythroid precursors by the ectopic expression of a dominant-negative form of FADD

Antisense targeting of 3' end elements involved in DUX4 mRNA processing is an efficient therapeutic strategy for facioscapulohumeral dystrophy: a new gene-silencing approach

Human adipose cells express CD4, CXCR4, and CCR5 receptors: a new target cell type for the immunodeficiency virus‐1?

Association Analysis Indicates That a Variant GATA-Binding Site in the PIK3CB Promoter Is a Cis-Acting Expression Quantitative Trait Locus for This Gene and Attenuates Insulin Resistance in Obese Children

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