A Promising Future for Stem-Cell-Based Therapies in Muscular Dystrophies—In Vitro and In Vivo Treatments to Boost Cellular Engraftment

iPSCs as a Platform for Disease Modeling, Drug Screening, and Personalized Therapy in Muscular Dystrophies

(2019) Jose Ortiz-Vitali et al.   Cells

MiR-27b Promotes Muscle Development by Inhibiting MDFI Expression

(2018) Lianjie Hou et al.   CELLULAR PHYSIOLOGY AND BIOCHEMISTRY

Untangling the complexity of limb-girdle muscular dystrophies

(2018) Teerin Liewluck et al.   MUSCLE & NERVE

Childhood-onset form of myotonic dystrophy type 1 and autism spectrum disorder: Is there comorbidity?

(2018) N. Angeard et al.   NEUROMUSCULAR DISORDERS

Inhibition of the Activin Receptor Type-2B Pathway Restores Regenerative Capacity in Satellite Cell-Depleted Skeletal Muscle

(2018) Luigi Formicola et al.   Frontiers in Physiology

High-Yield Purification, Preservation, and Serial Transplantation of Human Satellite Cells

(2018) Steven M. Garcia et al.   Stem Cell Reports

Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1

(2018) Marta López-Morató et al.   Frontiers in Neurology

The Unfolding Spectrum of Inherited Distal Myopathies

(2018) Margherita Milone et al.   MUSCLE & NERVE

Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy

(2018) Peggy P. Ho et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Prevalence, pathological mechanisms, and genetic basis of limb-girdle muscular dystrophies: A review

(2018) Eskandar Taghizadeh et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Rh-endostatin Concomitant with Chemotherapy Versus Single Agent Chemotherapy for Treating Soft Tissue and Bone Sarcomas: A Systematic Review and Meta-Analysis

(2018) Zhuo Ma et al.   JOURNAL OF PHARMACY AND PHARMACEUTICAL SCIENCES

Matrikines for therapeutic and biomedical applications

(2018) K. Sivaraman et al.   LIFE SCIENCES

Myotonic Dystrophies: Targeting Therapies for Multisystem Disease

(2018) Samantha LoRusso et al.   Neurotherapeutics

Facioscapulohumeral Muscular Dystrophy: Update on Pathogenesis and Future Treatments

(2018) Johanna Hamel et al.   Neurotherapeutics

Induced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and Therapy

(2018) Lubos Danisovic et al.   Cells

Fragments generated upon extracellular matrix remodeling: Biological regulators and potential drugs

(2017) Sylvie Ricard-Blum et al.   MATRIX BIOLOGY

CRISPR/Cas9-Induced (CTG⋅CAG) n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing

(2017) Ellen L. van Agtmaal et al.   MOLECULAR THERAPY

The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

(2017) S. Ryder et al.   Orphanet Journal of Rare Diseases

PABPN1 gene therapy for oculopharyngeal muscular dystrophy

(2017) A. Malerba et al.   Nature Communications

Regulation of myoblast differentiation by metabolic perturbations induced by metformin

(2017) Theodora Pavlidou et al.   PLoS One

Short-term ursolic acid promotes skeletal muscle rejuvenation through enhancing of SIRT1 expression and satellite cells proliferation

(2016) Nuredin Bakhtiari et al.   BIOMEDICINE & PHARMACOTHERAPY

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

(2016) Courtney S. Young et al.   Cell Stem Cell

Limb girdle muscular dystrophies

(2016) John Vissing   CURRENT OPINION IN NEUROLOGY

Emery-Dreifuss muscular dystrophy: the most recognizable laminopathy

(2016) Agnieszka Madej-Pilarczyk et al.   FOLIA NEUROPATHOLOGICA

Cellular Therapies for Muscular Dystrophies: Frustrations and Clinical Successes

(2016) Elisa Negroni et al.   HUMAN GENE THERAPY

Comparative effects of low-level laser therapy pre- and post-injury on mRNA expression of MyoD, myogenin, and IL-6 during the skeletal muscle repair

(2016) Agnelo Neves Alves et al.   LASERS IN MEDICAL SCIENCE

Duchenne muscular dystrophy

(2015) Eppie M Yiu et al.   JOURNAL OF PAEDIATRICS AND CHILD HEALTH

Evidence-based guideline summary: Evaluation, diagnosis, and management of congenital muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology and the Practice Issues Review Panel of the American Association of Neuromuscular & Electrodiagnostic Medicine

(2015) P. B. Kang et al.   NEUROLOGY

Regenerative medicine: Current therapies and future directions

(2015) Angelo S. Mao et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Stem Cell Differentiation Toward the Myogenic Lineage for Muscle Tissue Regeneration: A Focus on Muscular Dystrophy

(2015) Serge Ostrovidov et al.   Stem Cell Reviews and Reports

Stem Cell Differentiation Toward the Myogenic Lineage for Muscle Tissue Regeneration: A Focus on Muscular Dystrophy

(2015) Serge Ostrovidov et al.   Stem Cell Reviews and Reports

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

(2015) Hongmei Lisa Li et al.   Stem Cell Reports

Ex Vivo Expansion and In Vivo Self-Renewal of Human Muscle Stem Cells

(2015) Gregory W. Charville et al.   Stem Cell Reports

Matricryptins and matrikines: biologically active fragments of the extracellular matrix

(2014) Sylvie Ricard-Blum et al.   EXPERIMENTAL DERMATOLOGY

Wnt7a stimulates myogenic stem cell motility and engraftment resulting in improved muscle strength

(2014) C. Florian Bentzinger et al.   JOURNAL OF CELL BIOLOGY

Inhibition of JAK-STAT signaling stimulates adult satellite cell function

(2014) Feodor D Price et al.   NATURE MEDICINE

p38 MAPK signaling underlies a cell-autonomous loss of stem cell self-renewal in skeletal muscle of aged mice

(2014) Jennifer D Bernet et al.   NATURE MEDICINE

Prevalence of Muscular Dystrophies: A Systematic Literature Review

(2014) Alice Theadom et al.   NEUROEPIDEMIOLOGY

Duchenne and Becker Muscular Dystrophies

(2014) Kevin M. Flanigan   NEUROLOGIC CLINICS

Diagnostic approach to the congenital muscular dystrophies

(2014) Carsten G. Bönnemann et al.   NEUROMUSCULAR DISORDERS

Interleukin-6-induced satellite cell proliferation is regulated by induction of the JAK2/STAT3 signalling pathway through cyclin D1 targeting

(2013) M. Kurosaka et al.   CELL PROLIFERATION

Fibronectin Regulates Wnt7a Signaling and Satellite Cell Expansion

(2013) C. Florian Bentzinger et al.   Cell Stem Cell

Lying low but ready for action: the quiescent muscle satellite cell

(2013) Didier Montarras et al.   FEBS Journal

Muscular dystrophies

(2013) Eugenio Mercuri et al.   LANCET

Autologous Myoblast Transplantation for Oculopharyngeal Muscular Dystrophy: a Phase I/Iia Clinical Study

(2013) Sophie Périé et al.   MOLECULAR THERAPY

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

(2013) Rebecca J. Fairclough et al.   NATURE REVIEWS GENETICS

Distal myopathies

(2013) I. Pénisson-Besnier   REVUE NEUROLOGIQUE

Lamina-associated polypeptide (LAP)2α and nucleoplasmic lamins in adult stem cell regulation and disease

(2013) Kevin Gesson et al.   SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY

Collagen VI regulates satellite cell self-renewal and muscle regeneration

(2013) Anna Urciuolo et al.   Nature Communications

Laminin-111-derived peptides and cancer

(2013) Yamato Kikkawa et al.   Cell Adhesion & Migration

The Human Nuclear Poly(A)-Binding Protein Promotes RNA Hyperadenylation and Decay

(2013) Stefan M. Bresson et al.   PLoS Genetics

The ever-expanding spectrum of congenital muscular dystrophies

(2012) Eugenio Mercuri et al.   ANNALS OF NEUROLOGY

Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice

(2012) Radbod Darabi et al.   Cell Stem Cell

Stem cell therapies for muscle disorders

(2012) Francesco S. Tedesco et al.   CURRENT OPINION IN NEUROLOGY

Perspectives of stem cell therapy in Duchenne muscular dystrophy

(2012) Mirella Meregalli et al.   FEBS Journal

The myotonic dystrophies: molecular, clinical, and therapeutic challenges

(2012) Bjarne Udd et al.   LANCET NEUROLOGY

Proinflammatory Macrophages Enhance the Regenerative Capacity of Human Myoblasts by Modifying Their Kinetics of Proliferation and Differentiation

(2012) Maximilien Bencze et al.   MOLECULAR THERAPY

Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2

(2012) Richard J L F Lemmers et al.   NATURE GENETICS

Macrophages Improve Survival, Proliferation and Migration of Engrafted Myogenic Precursor Cells into MDX Skeletal Muscle

(2012) Pierre-François Lesault et al.   PLoS One

Wnt7a treatment ameliorates muscular dystrophy

(2012) J. von Maltzahn et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy

(2012) F. S. Tedesco et al.   Science Translational Medicine

The Muscular Dystrophies

(2012) Kevin Flanigan   SEMINARS IN NEUROLOGY

Systemic Delivery of Allogenic Muscle Stem Cells Induces Long-Term Muscle Repair and Clinical Efficacy in Duchenne Muscular Dystrophy Dogs

(2011) Karl Rouger et al.   AMERICAN JOURNAL OF PATHOLOGY

Antiprion drugs 6-aminophenanthridine and guanabenz reduce PABPN1 toxicity and aggregation in oculopharyngeal muscular dystrophy

(2011) Nicolas Barbezier et al.   EMBO Molecular Medicine

Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy

(2011) F. S. Tedesco et al.   Science Translational Medicine

Barx2 Is Expressed in Satellite Cells and Is Required for Normal Muscle Growth and Regeneration

(2011) Robyn Meech et al.   STEM CELLS

Myotonic Dystrophies 1 and 2: Complex Diseases with Complex Mechanisms

(2010) Benedikt Schoser et al.   CURRENT GENOMICS

Potential therapeutic applications of muscle-derived mesenchymal stem and progenitor cells

(2010) Wesley M Jackson et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells

(2010) Francesco Saverio Tedesco et al.   JOURNAL OF CLINICAL INVESTIGATION

Identification and characterization of a non-satellite cell muscle resident progenitor during postnatal development

(2010) Kathryn J. Mitchell et al.   NATURE CELL BIOLOGY

Substrate Elasticity Regulates Skeletal Muscle Stem Cell Self-Renewal in Culture

(2010) P. M. Gilbert et al.   SCIENCE

Mutations of the FHL1 Gene Cause Emery-Dreifuss Muscular Dystrophy

(2009) Lucie Gueneau et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Growth Factor Coinjection Improves the Migration Potential of Monkey Myogenic Precursors without Affecting Cell Transplantation Success

(2009) Jean-François Lafreniere et al.   CELL TRANSPLANTATION

Mesenchymal stem cells as anti-inflammatories: Implications for treatment of Duchenne muscular dystrophy

(2009) Thomas E. Ichim et al.   CELLULAR IMMUNOLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

In Vivo Myogenic Potential of Human CD133+ Muscle-derived Stem Cells: A Quantitative Study

(2009) Elisa Negroni et al.   MOLECULAR THERAPY

Further Characterisation of the Molecular Signature of Quiescent and Activated Mouse Muscle Satellite Cells

(2009) Viola F. Gnocchi et al.   PLoS One

Muscle stem cell behavior is modified by microRNA-27 regulation of Pax3 expression

(2009) C. G. Crist et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Highly Efficient, Functional Engraftment of Skeletal Muscle Stem Cells in Dystrophic Muscles

(2008) Massimiliano Cerletti et al.   CELL

Transcriptional targets of sirtuins in the coordination of mammalian physiology

(2008) Jerome N Feige et al.   CURRENT OPINION IN CELL BIOLOGY

Sirt1 increases skeletal muscle precursor cell proliferation

(2008) Christopher R. Rathbone et al.   EUROPEAN JOURNAL OF CELL BIOLOGY

Angiogenic laminin-derived peptides stimulate wound healing

(2008) Katherine M. Malinda et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Self-renewal and expansion of single transplanted muscle stem cells

(2008) Alessandra Sacco et al.   NATURE

Bone marrow stromal cells attenuate sepsis via prostaglandin E2–dependent reprogramming of host macrophages to increase their interleukin-10 production

(2008) Krisztián Németh et al.   NATURE MEDICINE

Cardiac assessment of limb–girdle muscular dystrophy 2I patients: An echography, Holter ECG and magnetic resonance imaging study

(2008) Karim Wahbi et al.   NEUROMUSCULAR DISORDERS

Pulmonary Passage is a Major Obstacle for Intravenous Stem Cell Delivery: The Pulmonary First-Pass Effect

(2008) Uwe M. Fischer et al.   STEM CELLS AND DEVELOPMENT

Clinical, molecular, and protein correlations in a large sample of genetically diagnosed Italian limb girdle muscular dystrophy patients

(2007) Michela Guglieri et al.   HUMAN MUTATION