x

Cerca un progetto di ricerca

* (*) campo obbligatorio

103 Risultato/i

Ordinato per

Finanziato da un ente associato a IRDiRC = Membro di una ERN =

Progetti di ricerca

BELGIO

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

CURE-DM1: In vivo CRISPR/Cas9-mediated correction of triplet nucleotide repeat expansion in DM1
VUB_ Vrije Universiteit Brussel, Campus Jette
Department of Gene Therapy and Regenerative Medicine

BELGIO

VLAAMS BRABANT
HEVERLEE (LEUVEN)

"3DMyoDM1": A novel human 3D in-vitro disease model for DM1
Laboratory of Bioengineering and Morphogenesis, KU Leuven
Laboratory of Bioengineering and Morphogenesis

CANADA

Ontario
OTTAWA, ONTARIO

Finanziato da un ente associato a IRDiRCNovel AMPK activators as relevant therapeutics for the treatment of Myotonic Dystrophy type 1 (DM1)
University of Ottawa
Department of Cellular & Molecular Medicine

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

CANADA

Québec
JONQUIÈRE

Finanziato da un ente associato a IRDiRCMotor, multisystemic and social participation assessment in myotonic dystrophy type 1 : a 9-year longitudinal study
CSSS - Centre de santé et de services sociaux de Jonquière
Centre de réadaptation Le Parcours

CANADA

Québec
QUÉBEC

Development of a treatment for Friedreich ataxia based on the administration of a TALE driving the expression of frataxin.
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Laboratoire de Génétique Humaine

CIPRO

Cyprus
ADDRESS: NOT PROVIDED - CY

Finanziato da un ente associato a IRDiRCLNA/2'OMe mixmers against toxic CUG expanded RNA
Institution: Information not provided - CY

CIPRO

Cyprus
NICOSIA

Finanziato da un ente associato a IRDiRCDevelopment of miRNA-based biomarkers for monitoring the progress in Myotonic Dystrophy type I
The Cyprus Institute of Neurology and Genetics
Molecular Genetics, Function & Therapy

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Finanziato da un ente associato a IRDiRCVenous thromboembolism in myotonic dystrophy type 1
Institution: Information not provided - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
PRAGUE

Finanziato da un ente associato a IRDiRCPreclinical assessment of clinic ready agents for the treatment of muscular dystrophy and spinal muscular atrophy
Faculty of Medicine and University Hospital Motol
Department of Pediatrics

FRANCIA

ILE-DE-FRANCE
EVRY

FRANCIA

ILE-DE-FRANCE
EVRY

Pathological modelling of Steinert Myotrophy using human embryonic stem cells carrying the causal mutation
GENOPOLE - Campus 1
I-Stem - Institut des cellules Souches pour le Traitement et l'Etude des maladies Monogéniques

FRANCIA

ILE-DE-FRANCE
EVRY

FRANCIA

ILE-DE-FRANCE
PARIS

Multidisciplinary Observational Study of Patients With Friedreich Ataxia
CHU Paris - Hôpital Robert Debré
Neurologie pédiatrique et maladies métaboliques

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCDM1 disease mechanisms in the central nervous system: from brain cell-specific pathogenesis to misregulated glutamate homeostasis
Centre de Psychiatrie et Neurosciences
Neurobiologie du vieillissement normal et pathologique

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCGene therapy in DM1 cells by induction of a TALE Nuclease
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCDM_Neuroglia : RNA toxicity and neuroglial miscommunication in myotonic dystrophy brains - FR
IMAGINE - Institut des Maladies Génétiques
Equipe "Triplets CTG instables et dystrophie myotonique"

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCHow to contract CAG.CTG repeats in myotonic dystrophy type 1
IMAGINE - Institut des Maladies Génétiques
Laboratoire "Triplets CTG instables et dystrophie myotonique"

FRANCIA

ILE-DE-FRANCE
PARIS

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCTricyclo-DNA antisense oligonucleotide treatment for Myotonic Dystrophy
Institut de Myologie - Hôpital Pitié-Salpêtrière
Équipe "Dystrophie Myotonique, Physiopathologie & Biothérapie"

FRANCIA

OCCITANIE
MONTPELLIER

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

Synthetic biology and Friedreich's Ataxia
CNRS Campus Joseph Aiguier
Laboratoire de Chimie Bactérienne (LCB) - UMR 7283

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

Finanziato da un ente associato a IRDiRCFRACOL: Synthetic biology to approach Friedreich ataxia - FR
CNRS Campus Joseph Aiguier
Laboratoire de Chimie Bactérienne (LCB) - UMR 7283

GERMANIA

Baden-Württemberg
HEIDELBERG

GERMANIA

Baden-Württemberg
ULM

Finanziato da un ente associato a IRDiRCGeNeRARe: German Network for RASopathies
Institute of Comparative Molecular Endocrinology der Universität Ulm
Cirstea Lab

GERMANIA

Bayern
ERLANGEN

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

GERMANIA

Hamburg
HAMBURG

Finanziato da un ente associato a IRDiRCGeNeRARe: German Network for RASopathies
UKE - Universitätsklinikum Hamburg-Eppendorf
Institut für Humangenetik

GERMANIA

Niedersachsen
GÖTTINGEN

Finanziato da un ente associato a IRDiRCGeNeRARe: German Network for RASopathies
Universitätsmedizin Göttingen
Klinik für Thorax-, Herz- und Gefäßchirurgie

GERMANIA

Nordrhein-Westfalen
DÜSSELDORF

Finanziato da un ente associato a IRDiRCGeNeRARe: German Network for RASopathies
Heinrich-Heine-Universität Düsseldorf
Institut für Biochemie und Molekularbiologie II

GERMANIA

Sachsen-Anhalt
MAGDEBURG

Finanziato da un ente associato a IRDiRCGeNeRARe: German Network for RASopathies
Otto-von-Guericke-Universität Magdeburg
Abteilung Genetik & Molekulare Neurobiologie

GIAPPONE

JAPAN
MIYAGI

Finanziato da un ente associato a IRDiRCPathogenesis and novel therapeutic approarches in RASoapthies
Tohoku University School of Medicine
Department of Medical Genetics

GIAPPONE

JAPAN
OSAKA

Finanziato da un ente associato a IRDiRCEvidence-generating clinical research for myotonic dystrophy
Osaka University Graduate School of Medicine
Department of Functional Diagnostic Science

ITALIA

LAZIO
ROMA

Caratterizzazione clinica e molecolare di sindromi genetiche con cardiopatie congenite
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
U.O.C. Laboratorio di Genetica Medica

ITALIA

LAZIO
ROMA

Determinanti molecolari delle malattie neurodegenerative
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
Unità di Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare

ITALIA

LOMBARDIA
CUSANO MILANINO

SPAGNA

Andalucía
CÁDIZ

Finanziato da un ente associato a IRDiRCCell-type-specific biomarkers in peripheral fluids in trinucleotide diseases
Hospital Universitario Puerta del Mar
Instituto de Investigación e Innovación en Ciencias Biomédicas de Cádiz

SPAGNA

Cataluña
BADALONA

Finanziato da un ente associato a IRDiRCPre-clinical evaluation of a new gene therapy vector based on adeno-associated virus in the reversal of the neurological signs in an acute disease mouse model of Friedreich's Ataxia
Instituto de Investigación Germans Trias i Pujol
Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol

SPAGNA

Cataluña
BADALONA

Finanziato da un ente associato a IRDiRCDIMINUTES, Childhood and adult myotonic dystrophy: evaluation of new treatments and pathogenicity through genetic, epigenetic and molecular imaging analysis
Instituto de Investigación Germans Trias i Pujol
Grupo de investigación en neuromuscular y neuropediátrica

SPAGNA

Comunidad Valenciana
VALENCIA

Finanziato da un ente associato a IRDiRCComprehensive genomic characterization of hereditary myeloid syndromes
IIS La Fe - Instituto de Investigación Sanitaria La Fe
Grupo de investigación en hematología y hemoterapia

SPAGNA

Comunidad Valenciana
VALENCIA

Genomic edition of the FXN Gene through the CRISPR / Cas9 system in lymphocytes of patients with Friedreich's ataxia
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en fisiopatología celular y orgánica del estrés oxidativo

SPAGNA

Comunidad Valenciana
VALENCIA

Validation of circulating microRNAs as prognostic biomarkers and clinical evolution in Friedreich ataxia. Towards precision medicine in Rare Diseases
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en fisiopatología celular y orgánica del estrés oxidativo

SPAGNA

Comunidad Valenciana
VALENCIA

Finanziato da un ente associato a IRDiRCNew Therapies in Friedreich Ataxia
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en fisiopatología celular y orgánica del estrés oxidativo

SPAGNA

Comunidad Valenciana
VALENCIA

Finanziato da un ente associato a IRDiRCPreclinical development of an innovative drug for myotonic dystrophy
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en genómica traslacional

SPAGNA

Comunidad Valenciana
VALENCIA

ARTHEx biotech: anti-microRNAs for the treatment of myotonic dystrophy
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en genómica traslacional

SPAGNA

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

SPAGNA

País Vasco
BARAKALDO

Finanziato da un ente associato a IRDiRCMyoblots for rapid evaluation of new treatments for myotonic dystrophy (MaTILDa)
Instituto de Investigación sanitaria Biocruces Bizkaia
Grupo de investigación en enfermedades neuromusculares

STATI UNITI

South Dakota
SOUTH DAKOTA

Finanziato da un ente associato a IRDiRCAberrant mitochondrial redox regulation in Friedreich's Ataxia
Sanford Research/University of South Dakota
VITIELLO LAB- Depatment of Pediatrics

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCRna toxicity and muscle regeneration
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCEpigenetic mechanisms regulating the igf2/h19 and kcnq1 locus
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMammalian iron-sulfur cluster biogenesis
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCOpposing pathways in mammalian sex determination
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

SVEZIA

Region Stockholm
HUDDINGE

The functional organisation of the brain
Karolinska Institutet - Huddinge
Department of Clinical Neuroscience

SVEZIA

Region Västerbotten
UMEÅ

SVIZZERA

Suisse Alémanique
BERN

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCPredictive genetic testing in hereditary cardiac diseases: Evaluation of psycho-social impact and multidisciplinary management
APHP.Sorbonne Université, Hôpital de la Pitié-Salpêtrière
Départements de génétique et de cardiologie - Centre de référence des maladies cardiaques héréditaires

FRANCIA

ILE-DE-FRANCE
PARIS

GIAPPONE

JAPAN
TOKYO

Finanziato da un ente associato a IRDiRCDevelopment of novel disease-causing gene based therapies for cardiomyopathies
Tokyo Medical and Dental University Medical Research Institute

REGNO UNITO; GRAN BRETAGNA

Hampshire
SOUTHAMPTON

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCPersonalizing The Risk Of Stroke And Silent Brain Infarct In Cardiovascular Disease
Hospital General Universitario Gregorio Marañón
Servicio de Cardiología

CANADA

Ontario
TORONTO

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Finanziato da un ente associato a IRDiRCMechanosensitive ionic channels in zebrafish heart regeneration
Institution: Information not provided - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCSTaHR : Stimulation of Targeted Homologous Recombination for Gene Therapy - FR
Muséum National d'Histoire Naturelle
Laboratoire Structure et Instabilité des Génomes - INSERM U1154 - CNRS 7196

FRANCIA

PAYS DE LA LOIRE
NANTES

Finanziato da un ente associato a IRDiRCFROGH : FRench Regional Origins in Genetics for Health - FR
Institut de Recherche en Santé - Université de Nantes
L'Institut du thorax - Inserm UMR 1087 / CNRS UMR 6291

GIAPPONE

JAPAN
TOKYO

Finanziato da un ente associato a IRDiRCFormation of integrated omics analysis base of refractory diseases in children and perinatal period
National Center for Child Health and Development
Research Institute

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCA powerful web-based discovery platform for rare disease genetics
Institution: Information not provided - US

Progetti di ricerca multicentrici