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140 Resultaat/Resultaten
Gefinancierd door een IRDiRC-lid = Member of a ERN =
Onderzoeksprojecten

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
SIX homeoproteins and muscle stem cells properties
Institution: Information not provided - FR

Ontario
HAMILTON
Role of Xin, an actin-binding protein, in satellite cells and muscular dystrophies
McMaster University
Department of Pathology and Molecular Medicine

Baden-Württemberg
HEIDELBERG
Cellular mechanisms leading to desminopathy: Segregation, aggregation and proteostasis imbalance of desmin mutants in muscle cells and tissue
Deutsches Krebsforschungszentrum
Arbeitsgruppe Funktionelle Zellarchitektur (B065)

Bayern
ERLANGEN
Cellular mechanisms leading to desminopathy: Segregation, aggregation and proteostasis imbalance of desmin mutants in muscle cells and tissue
Universitätsklinikum Erlangen - Kopfkliniken
Neuropathologisches Institut

Bayern
ERLANGEN
Neuromuscular endplate pathology in autosomal dominant and recessive desminopathies
Universitätsklinikum Erlangen - Kopfkliniken
Neuropathologisches Institut

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Characterization of Liver-induced allo-specific CD8 regulatory T lymphocytes
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Molecular mechanisms of chronic muscle pain - Focus on Acid-Sensing Ion Channels (ASICs)
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
A new mouse model for desminopathies: physiopathological mechanisms, muscle repair and therapy
Institution: Information not provided - FR

BRETAGNE
RENNES
Structure and function of dystrophin in relation with mutations / deletions in the human dystrophinopathies
Faculté de médecine de Rennes
Institut de Génétique et Développement de Rennes - UMR6290

ILE-DE-FRANCE
EVRY
Consolidating data for rescue of missense mutations from degradation
Généthon
Centre de Recherche Généthon

ILE-DE-FRANCE
FONTENAY-AUX-ROSES
Functional analysis of separase-dependent lamins' regulation in AD-EDMD
Institut de biologie François Jacob
Laboratoire Réparation Et Vieillissement - LREV

ILE-DE-FRANCE
PARIS
Treatment of Anthacycline-Induced Cardiomyopathy by Intravenous Administration of Cardiovascular Progenitor Cell-Derived Extracellular Vesicles
CHU Paris IdF Ouest - HEGP Hôpital Européen Georges Pompidou
Thérapie cellulaire en pathologie cardiovasculaire

ILE-DE-FRANCE
PARIS
Caveolin-assisted sphingolipid transport to the plasma membrane in epidemiology of muscle dystrophies
CLCC Institut Curie
Institut Curie

ILE-DE-FRANCE
VERSAILLES
The impact of residual dystrophin on the natural history of dystrophinopathies.
Université de Versailles Saint-Quentin
UMR U1179 - Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées

OCCITANIE
MONTPELLIER
Unravelling molecular mechanisms of DMD gene splicing regulation and their roles as disease modifiers in Duchenne muscular Dystrophy.
Institut Universitaire de Recherche Clinique EA 7402 Université de Montpellier
Laboratoire de Génétique de Maladies Rares

PROVENCE-ALPES-COTE D'AZUR
NICE
Deciphering the role of the heterochromatin conformation in Emery Dreifuss Muscular Dystrophy (EDMD)
Institute for Research on Cancer and Aging
Department of Medical Genetics

ISRAEL
ADDRESS: NOT PROVIDED - IL
Epigenetic analysis of myonuclei defective in nuclear envelope components in Drosophila muscles as a model for studying Emery-Dreifuss muscular dystrophy
Institution: Information not provided - IL

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Bone phenotype in Duchenne muscular dystrophy: unveiling the role of LCN2 and implications for therapy.
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
PGC1 alpha gene expression regulation and Mitochondrial Biogenesis impairment in Muscular Dystrophies: new molecular signatures for novel therapeutic strategy
Institution: Information not provided - IT

LAZIO
ROMA
Role of miR-200c in dystrophic muscle regeneration of mdx mice and DMD patients
Istituto Dermopatico dell'Immacolata - IRCCS
I Divisione Dermatologia

LAZIO
ROMA
In vivo characterization of miR-200c in regeneration of dystrophic skeletal muscles in mdx mice
Sapienza Università di Roma
Dipartimento di Genetica e Biologia Molecolare

LIGURIA
GENOVA
Extracellular ATP and T regulatory cells: new therapeutics targets in alpha-sarcoglycan deficient muscular dystrophy (LGMD2D)
IRCCS Istituto G. Gaslini - Ospedale Pediatrico
U.O.C. Neurologia pediatrica e Malattie muscolari

LOMBARDIA
MILANO
Dissecting the role of chromatin structure and alternative splicing in the development and pathology of Emery-Dreifuss Muscular Distrophy
IFOM - Istituto FIRC di Oncologia Molecolare
Firc Institute of Molecular Oncology (IFOM)

Cataluña
BARCELONA
Characterization of the role of fibroadipogenic precursors in the degenerative process of muscular dystrophies
Hospital de la Santa Creu i Sant Pau
Unidad funcional de enfermedades autoinmunitarias sistémicas

Comunidad Valenciana
VALENCIA
Cognitive implications in Duchenne/Becker dystrophies. Spanish cohort
IIS La Fe - Instituto de Investigación Sanitaria La Fe
Grupo de investigación en patología neuromuscular y ataxias

Comunidad Valenciana
VALENCIA
Molecular mechanisms in the development of scoliosis in limb-girdle muscular dystrophies
Universitat de València. Facultat de Medicina i Odontologia
Departamento de Bioquímica y Biología Molecular

Washington
ADDRESS: NOT PROVIDED - US
Interventions in genetic counseling
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Mechanosignaling functions of the dystrophin glycoprotein complex in muscular dystrophy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
The sarcoglycan complex in skeletal muscle mechanotransduction
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Project 1: preclinical development of surrogate gene therapy using galgt2
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Regulation of subcellular organization in skeletal muscle
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Safety and feasibility study of transvenous limb perfusion with normal saline in
Institution: Information not provided - US

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
NEXTGEN-AAV: Development of next-generation AAV vectors for Duchenne muscular dystrophy
VUB_ Vrije Universiteit Brussel, Campus Jette
Department of Gene Therapy and Regenerative Medicine

ARRONDISSEMENT BRUSSELS-CAPITAL
LOUVAIN-LA-NEUVE
Therapeutic potential of AdipoRon, an Adiponectin receptor agonist, in Duchenne Muscular Dystrophy
Université Catholique de Louvain - UCL
Université catholique de Louvain

Alberta
EDMONTON
Targeted delivery of antisense-mediated exon skipping therapy in transgenic pig model of DMD
University of Alberta
Department of Medical Genetics

Ontario
OTTAWA
Genetic Regulation of Myogenesis
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research

Ontario
OTTAWA
Stimulating cilia-mediated Hedgehog signaling to restore dystrophin-deficient satellite cell function
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research

Ontario
OTTAWA, ONTARIO
Post-transcriptional Regulation of Utrophin in Skeletal Muscle: Implications for New Therapeutic Strategies for Duchenne Muscular Dystrophy
University of Ottawa
Faculty of Medicine -

Ontario
TORONTO
Growth arrest and osteoporosis in Duchenne muscular dystrophy patients treated with glucocorticoids
Mount Sinai Hospital
Lunenfeld-Tanenbaum Research Institute

Québec
ADDRESS: NOT PROVIDED - CA
Elucidation of Wnt7a mechanism of action for muscle regeneration
Institution: Information not provided - CA

Québec
ADDRESS: NOT PROVIDED - CA
Targeting of Histone Deacetylase 6 (HDAC6) in Neuromuscular Diseases
Institution: Information not provided - CA

Québec
ADDRESS: NOT PROVIDED - CA
Repurposing drugs that target eEF1A2 to increase translation of utrophin in dystrophic muscle
Institution: Information not provided - CA

Québec
MONTRÉAL
Strategies for therapy of respiratory muscle failure in muscular dystrophy
Meakins-Christie Laboratories
Meakins-Christie Labs

Québec
QUÉBEC
Correction of the dystrophin gene with TAL effector nuclease and the CRISPR system
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Unité de Génétique humaine, Axe Neurosciences

Québec
QUÉBEC
Can laminin-111 be used to treat Duchenne Muscular Dystrophy alone or in combination with myoblast transplantation?
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Unité de Génétique humaine, Axe Neurosciences

Bayern
ERLANGEN
Progression study on the characterization of skeletal muscles by magnetic resonance imaging in Duchenne muscular dystrophy
ZSEER - Zentrum für Seltene Erkrankungen Erlangen

Berlin
BERLIN
TAMDMD: Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial - DE
DRK Kliniken Berlin Westend
Klinik für Kinder- und Jugendmedizin

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Gene transfer tolerance in combined liver and muscle rAAV gene therapy
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Validation of non-invasive non-volitional methods for monitoring respiratory muscle function in Duchenne muscular dystrophy
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Translational suppression of nonsense mutations found in DMD
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Clinical Implementation of Noninvasive Prenatal Testing for Duchenne Muscular Dystrophies
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Human reserve cells: a dynamic tool to discover genes involved in muscle stem cell quiescence
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Liver Gene Transfer-Induced allogeneic Tolerance for organ transplantation and cell therapy
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Characterization of CASK protein function in the regulation of ion channels in cardiomyocytes and its role in cardiac electrophysiology in vivo
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Characterization of a new model of mdx mice deficient in the enzyme CD38: towards the protective role on the Ca2+ homeostasis deregulation
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Dystrophin-deficient diaphragm dysfunction induced by mechanical ventilation in mdx mice.
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Involvement of endo/sarcoplasmic reticulum - mitochondria interaction in the Duchenne muscular dystrophy
Institution: Information not provided - FR

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Dystrophin and neuronal plasticity: towards a possible treatment of the cognitive defects in DMD using novel antisense oligonucleotides
Institution: Information not provided - FR

ILE-DE-FRANCE
GIF-SUR-YVETTE
Rescue_ribosome : Rescue Pathways for unrecycled ribosomes - FR
Institut de Biologie Intégrative de la Cellule (I2BC)
Département Biologie des Génomes

ILE-DE-FRANCE
GIF-SUR-YVETTE
Restoration of Ca2+-signalling in mdx mice by targeting the endo-lysosmal two-pore channel (TPC)
Institut des Neuroscieces Paris-Saclay
Code neuronal & perception auditive

ILE-DE-FRANCE
PARIS

ILE-DE-FRANCE
PARIS
Implementation of non-invasive prenatal diagnosis of rare and severe monogenic diseases
CHU Paris Centre - Hôpital Cochin, Site Cochin
Service de génétique et biologie moléculaires

ILE-DE-FRANCE
PARIS
Induced pluripotent stem cells-based strategies and disease modeling to unravel signaling-induced epigenetic networks in healthy and in Duchenne muscle dystrophic muscles: the TGF beta/ Wnt regulated histone lysine methylome
CNRS - Centre national de la recherche scientifique
Centre National de la Recherche Scientifique

ILE-DE-FRANCE
PARIS
The impact of cellular senescence and in vivo reprogramming in Duchen-Muscular Dystrophy
Institut Pasteur

ILE-DE-FRANCE
VERSAILLES
Induction of immunological tolerance by dual muscle and liver gene transfer for Duchenne muscular dystrophy
Université de Versailles Saint-Quentin
UMR U1179 - Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées

ILE-DE-FRANCE
VERSAILLES
Elucidate the cellular and molecular mechanisms involved in the generation of revertant dystrophin-positive fibers using the dystrophic DmdEGFP-mdx reporter mouse.
Université de Versailles Saint-Quentin
UMR U1179 - Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées

ILE-DE-FRANCE
VERSAILLES
Elucidate the molecular mechanism leading to the generation of revertant dystrophin-positive fibers in muscle dystrophic DmdEGFP-mdx reporter mouse
Université de Versailles Saint-Quentin
Laboratoire Handicap neuromusculaire : Physiopathologue, Biotechnologies et Pharmacologies appliquées (END-ICAP) - UMR U1179

OCCITANIE
MONTPELLIER
Towards a better understanding of the interplay between cis-acting elements and trans factors driving DMD pre-MRNA splicing
IURC - Institut Universitaire de Recherche Clinique
Laboratoire de génétique moléculaire

OCCITANIE
MONTPELLIER
Cardiac dysfunction in Duchenne Muscle Dystrophy children : pathophysiological role of type 2 ryanodine receptor through hiPSC-derived cardimyocytes
Université Montpellier II
Caractérisation de cardiomyocytes DMD et CPVT « patient-specific » dérivés de cellules souches pluripotentes induites

PAYS DE LA LOIRE
NANTES
TOLGEN: In vivo AAV-based gene transfer to the muscle: Deciphering host immune responses - FR
IRS2 - Nantes Biotech
Thérapie Génique Translationnelle des Maladies Génétiques

County Cork
CORK
Monoclonal xIL-6R antibodies as a treatment for memory dysfunction in the mdx mouse model of Duchenne muscular dystrophy
University College Cork
Department of Physiology

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Pin1: a new potential target to induce slow fiber conversion in Duchenne Muscular Dystrophy
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Immune system involvement in DMD pathology: the role of the immunoproteasome and its therapeutic potential.
Institution: Information not provided - IT

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Therapeutic relevance of the sphingolipid-metabolizing enzyme acid sphingomyelinase as a new pathogenetic factor in Duchenne muscular dystrophy
Institution: Information not provided - IT

LAZIO
ROMA
Exosome-mediated HDACi/miR-143/STAT3 network in the regulation of satellite cells expansion and muscle regeneration
IRCCS Fondazione Santa Lucia
Laboratorio di Epigenetica e Farmacologia Rigenerativa

LAZIO
ROMA
New therapeutic strategies based on FAPs-derived Exosomes in the treatment of Duchenne Muscular Dystrophy
IRCCS Fondazione Santa Lucia
Laboratorio di Epigenetica e Farmacologia Rigenerativa

LAZIO
ROMA
Role of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

LAZIO
ROMA
RNA-based studies of Duchenne Muscular Dystrophy: post-transcriptional control and role of non coding RNAs in normal and dystrophic muscle development
Sapienza Università di Roma
Dipartimento di Biologia e Biotecnologie "Charles Darwin"

LOMBARDIA
MILANO
HMGB1 Redox Forms as New Targets in Duchenne Muscular Dystrophy
IRCCS Ospedale San Raffaele
Unit of Chromatin Dynamics - division of Genetics and Cell Biology

VENETO
PADOVA
Pre-clinical evaluation of biocompatible nanoparticles as delivery system of 20-methyl-phosphorothioate antisense oligoribonucleotides for exon skippin- mediated dystrophin in restoration
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Istologia, Microbiologia e Biotecnologie Biomediche

VENETO
PADOVA
Role of Cyclophilins in Duchenne Muscular Dystrophy
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Scienze Biomediche

VENETO
PADOVA
Targeting the Mitochondrial Calcium Uniporter to counteract Duchenne Muscular Dystrophy
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Scienze Biomediche

VENETO
TREVISO
Cell therapy for muscle dystrophies: delivery of myogenic precursor cells and amniotic stem cells via polymeric scaffolds
Azienda ULSS 9 - Ospedale di Treviso
Chirurgia Pediatrica

JAPAN
NAGANO
Research on natural history of Becker muscular dystrophy for establishment of preventive medicine
National Hospital Organization Matsumoto Medical Center
Department of Neurology

JAPAN
OKAYAMA
Research for practical application of an innovative peptide drug for DMD
Kawasaki Medical School
Department of Neurology

JAPAN
TOKYO
Development of novel RNA medicines for treating muscular diseases using microRNAs capable of inducing muscular differentiation and regeneration
National Center of Neurology and Psychiatry
Department of Molecular Pharmacology

JAPAN
TOKYO
Development of a novel peptide-conjugated phosphorodiamidate morpholino therapy for Duchenne muscular dystrophy.
National Center of Neurology and Psychiatry
Department of Molecular Therapy

Gelderland
NIJMEGEN
Functional arm training: study for boys with Duchenne Muscular Dystrophy
Radboudumc - Oost
Afdeling Revalidatie

Gelderland
NIJMEGEN
Development of an exoskeleton for the arms for boys with Duchenne Muscular Dystrophy
Radboudumc - Oost
Afdeling Revalidatie

Zuid-Holland
LEIDEN
Weten vrouwen uit families met een Duchenne patiënt dat ze draagster kunnen zijn en ook een zoon met deze ziekte kunnen krijgen?
LUMC - Leids Universitair Medisch Centrum
Laboratorium voor Diagnostische Genoomanalyse

Zuid-Holland
LEIDEN
Weten vrouwen uit families met een Duchenne patiënt dat ze draagster kunnen zijn en ook een zoon met deze ziekte kunnen krijgen?
LUMC - Leids Universitair Medisch Centrum
Afdeling Klinische Genetica

WIEN
WIEN
Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care - AT
Gottfried von Preyer'sches Kinderspital
Abteilung für Kinder- und Jugendheilkunde

Cataluña
BARCELONA
Novel strategies to ameliorate Duchenne Muscular Dystrophy
Universitat Pompeu Fabra. Campus del Mar
Grupo de Biología Celular

Florida
MIAMI
whole body periodic acceleration a novel treatment for duchenne cardiomyopathy in mdx mice
Mount Sinai Medical Center

Massachusetts
BOSTON
Discovery of small-molecules modulating new modifier genes in Duchenne Muscular Dystrophy: a novel and promising therapy to escape the dystrophic phenotype
Boston Children's Hospital

Massachusetts
BOSTON
Discovery of small-molecules modulating new modifier genes in Duchenne Muscular Dystrophy: a novel and promising therapy to escape the dystrophic phenotype.
Boston Children's Hospital

Washington
ADDRESS: NOT PROVIDED - US
STAT3 signaling network in MuSCs as therapeutic target for DMD
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Use of a novel in vitro DMD model to study muscle fusion during health and disease
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Whole body single aav microgene therapy in canine dmd
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Safety and efficacy of systemic gene therapy in informative models for dmd
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Therapeutics for rare and neglected diseases - science
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
A drug based approach for integrin-mediated alleviation for muscular dystrophy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Gene therapy platform for rare diseases
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Crispr/cas9-based gene editing for the correction of duchenne muscular dystrophy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Exploring the role of fbxw7 in regulating satellite cell function and skeletal muscle regeneration
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Innovative approaches to treat duchenne muscular dystrophy using ipsc-derived muscle progenitors
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Interrogating functional and molecular properties of pax7+ putative skeletal muscle stem/progenitor cells derived from human ipscs of healthy donors and duchenne muscular dystrophy patients
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Microtubule regulated mechanotransduction in skeletal muscle
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Mr assessment of bioenergetics and microvascular function in dystrophic muscle
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Multiscale modeling for treatment discovery in duchenne muscular dystrophy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Muscle tregs in health and disease
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Sarcolipin in duchenne muscular dystrophy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Targeting leukemia inhibitory factor to dystrophic muscle via a macrophage-specific transgene
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Treatment of duchenne muscular dystrophy with the muscle calcium pump
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Assessing and modulating inflammation and fibrosis in dystrophic muscle
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Bioinformatics and genomics
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Highthroughput screening and cell repository
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Optimization of ao drugs 45; 51 & 53
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Translational and pre-clinical studies of aav-mediated gene therapy of muscular
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Biomarker discovery for ao accumulation in kidney
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Clinical evaluation of urine biomarkers for morpholino
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Coupling molecular and clinical phenotypes with ipsc disease modeling for myoediting of duchenne muscular dystrophy
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Correction of muscular dystrophy in mice by crispr/cas9-mediated genomic editing
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Preclinical dosing optimization: dosing schedule; tissue
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Strategies to overcome immunity in gene therapy of dmd
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
The implications of dystrophin-specific t cells for dmd gene correction
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Pediatric toxicity and efficacy in long-term systemic treatment with anti-sense
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Functional characterization and therapeutic implications of a dystrophin isoform harboring a deletion in the dystrophin actin binding domain 1
Institution: Information not provided - US

Washington
ADDRESS: NOT PROVIDED - US
Prospective Study in Cardiac Disease in Duchenne Muscular Dystrophy (DMD)
Institution: Information not provided - US

Edinburgh
EDINBURGH
Characterisation of skeletal development and the use of anabolic agents in murine models of Duchenne muscular dystrophy
University of Edinburgh
The Roslin Institute

Oxfordshire
OXFORD
MICA: The role of utrophin in DMD and its therapeutic potential
University of Oxford
Department of Physiology, Anatomy and Genetics
Multicentrische onderzoeksprojecten
- Ottawa Hospital Research Institute
- Sprott Center for Stem Cell Research
- Zentrum für Kinder- und Jugendmedizin Freiburg
- Klinik für Neuropädiatrie und Muskelerkrankungen
- Institut de Biologie Intégrative de la Cellule (I2BC)
- Département Biologie des Génomes
- Université Pierre et Marie Curie - Paris 6
- Groupe Myologie - UMR S 787
- IRS2 - Nantes Biotech
- Thérapie Génique Translationnelle des Maladies Génétiques
- A.O.U. Sant'Anna - Polo Chimico-Bio-Medico
- U.O.C. di Genetica Medica
- IRCCS Ospedale San Raffaele
- Istituto di Ricerca per le Cellule Staminali
- Newcastle upon Tyne Hospitals NHS Trust
- John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine
- Universitäts-Kinderspital beider Basel UKBB
- Die Abteilung für Neuro- und Entwicklungspädiatrie

Ontario
OTTAWA
SIRD: Stimulating Intrinsic Repair for DMD

Baden-Württemberg
FREIBURG
CARE-NMD : Improving care for Duchenne muscular dystrophy

ILE-DE-FRANCE
GIF-SUR-YVETTE
Rescue_ribosome : Rescue Pathways for unrecycled ribosomes

ILE-DE-FRANCE
PARIS
ENDOSTEM: Activation of vasculature associated stem cells and muscle stem cells for the repair and maintenance of muscle tissue

PAYS DE LA LOIRE
NANTES
TOLGEN: In vivo AAV-based gene transfer to the muscle: Deciphering host immune responses

EMILIA ROMAGNA
FERRARA
BIO-NMD: identifying and validating pre-clinical biomarkers for diagnostics and therapeutics of neuromuscular disorders

LOMBARDIA
MILANO
OptiStem: Optimisation of stem cell therapy for clinical trials of degenerative skin and muscle diseases (TERMINATED)

Tyne & Wear
NEWCASTLE UPON TYNE
BIOIMAGE-Neuromuscular Diseases

Suisse Alémanique
BASEL