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Progetti di ricerca

CANADA

Ontario
TORONTO

AUSTRIA

STEIERMARK
GRAZ

CRISPR/Cas9 genome engineering to dissect MPN (myeloproliferative neoplasm) pathogenesis
Medizinische Universität Graz
Klinische Abteilung für Hämatologie

FRANCIA

ILE-DE-FRANCE
FONTENAY-AUX-ROSES CEDEX

Identification by high-throughput functional screens of Fanconi disease modifying genes
DRF/Institut François-Jacob CEA Paris-Saclay
Laboratoire d'Exploration Fonctionnelle des Génomes - LEFG

FRANCIA

NOUVELLE AQUITAINE
BORDEAUX

GERMANIA

Berlin
ADDRESS: NOT PROVIDED - DE

GERMANIA

Sachsen-Anhalt
HALLE (SAALE)

ITALIA

EMILIA ROMAGNA
MELDOLA

Integrazione multiomica e caratterizzazione multilivello dei disordini e delle neoplasie maligne ematologiche
IRST - Istituto Scientifico Romagnolo per lo studio e la cura dei Tumori
IRCCS Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori

ITALIA

LOMBARDIA
MILANO

RBDD: Rede Database delle Malattie Rare della Coagulazione (coordination)
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Laboratorio di Patologie Molecolari Applicate alla Clinica

SVIZZERA

Suisse Alémanique
BASEL

Next generation advanced therapies for fight Beta-hemoglobinopathies via rational intervention in beta-globin regulatory network
Departement Biosysteme (D-BSSE)
Departement für Biosysteme und Ingenieurwissenschaften ETH Zürich

FRANCIA

ILE-DE-FRANCE
PARIS

Constitutive hematologic diseases: clinical and therapeutic research, molecular biology
GHU AP-HP Nord. Université de Paris - Hôpital Saint-Louis
Service d'hématologie pédiatrique

PORTOGALLO

SUL
LISBOA

Hemoglobinopathies molecular research
Instituto Nacional de Saúde Dr. Ricardo Jorge - Lisboa
Departamento de Genética

AUSTRIA

WIEN
ADDRESS: NOT PROVIDED - AT

AUSTRIA

WIEN
WIEN

Finanziato da un ente associato a IRDiRCRIBOEUROPE: The European Ribosomopathy Consortium - AT
AKH BT 25.3
Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases

AUSTRIA

WIEN
WIEN

Genetic screening to identify suppressors of Diamond-Blackfan anemia
Vienna Biocenter
IMBA - Institute of Molecular Biotechnology

BELGIO

HAINAUT
GOSSELIES

Finanziato da un ente associato a IRDiRCRIBOEUROPE: The European Ribosomopathy Consortium - BE
Biopark campus
The 'RNA Metabolism' Lab - Université Libre de Bruxelles (ULB)

BELGIO

HAINAUT
GOSSELIES

Finanziato da un ente associato a IRDiRCDBAGenCure: Lentiviral-mediated gene therapy for Diamond Blackfan Anemia: Preclinical Safety and Efficacy Studies - BE
Biopark campus
The 'RNA Metabolism' Lab - Université Libre de Bruxelles (ULB)

CANADA

Ontario
TORONTO

Finanziato da un ente associato a IRDiRCPathobiology and robosomal insufficiency in Shwachman-Diamond syndrome
Hospital for Sick Children, Research Institute

COREA DEL SUD

KOREA, REPUBLIC OF
SEOUL

FINLANDIA

Finland
HELSINKI

Finanziato da un ente associato a IRDiRCGenomic editing for treating hemoglobin diseases
University of Helsinki
Stem Cells and Metabolism Research Program

FRANCIA

GRAND-EST
STRASBOURG

FRANCIA

ILE-DE-FRANCE
LE KREMLIN-BICÊTRE

Diagnosis and treatment of acquired hemostatic disorders due to auto-antibodies
APHP. Université Paris-Saclay, Hôpital Bicêtre
Service d'Hématologie

FRANCIA

ILE-DE-FRANCE
PARIS

Physiopathology of human myelofibrosis with myeloid metaplasia
AP-HP.Sorbonne Université - Hôpital Saint-Antoine
UF de cytogénétique onco-hématologique

FRANCIA

ILE-DE-FRANCE
PARIS

Non-invasive prenatal diagnosis of sickle cell anemia by next generation sequencing (NGS)
CHU Paris - Hôpital Robert Debré
UF de Génétique Moléculaire

FRANCIA

ILE-DE-FRANCE
PARIS

Microcephaly, Fanconi Anemia and Praxial Disorders (MicroFancII)
CHU Paris - Hôpital Robert Debré
UF de Génétique clinique

FRANCIA

ILE-DE-FRANCE
PARIS

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCRIBOEUROPE: The European Ribosomopathy Consortium - FR
Faculté de Médecine Paris Diderot Paris 7 - site Bichat
Centre de Recherche sur l'Inflammation (UMR 1149 Inserm - Université Paris Diderot - ERL CNRS 8252)

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - FR
GHU AP-HP Nord. Université de Paris - Hôpital Saint-Louis
Laboratoire d'hématologie

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCCRISPR/Cas9 mediated induction of fetal hemoglobin synthesis for the treatment of beta-hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCDevelopment of Innovative Therapeutic Strategies for Beta-Hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCIA

ILE-DE-FRANCE
PARIS

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCDevelopment of a universal prime editing approach to beta-hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCIA

ILE-DE-FRANCE
PARIS

Collaborative biological research for childhood Evans syndrome (ES) and Autoimmune hemolytic anemia (AIHA)
Institut National de la Transfusion Sanguine (INTS)
Physiologie du globule rouge normal et pathologique

FRANCIA

ILE-DE-FRANCE
VILLEJUIF

Physiopathology of human myelofibrosis with myeloid metaplasia
GHU Paris-Sud - Hôpital Paul Brousse
Les cellules souches: de leurs niches à leurs applications thérapeutiques

FRANCIA

OCCITANIE
TOULOUSE

Finanziato da un ente associato a IRDiRCRIBODBA: Blackfan-Diamond anemia: molecular mechanisms of a ribosomal disease
Université Paul Sabatier
Laboratoire de Biologie Moléculaire Eucaryote - UMR 5099

FRANCIA

OCCITANIE
TOULOUSE

Finanziato da un ente associato a IRDiRCDEVDBA: Ontogeny as a critical determinant of DBA sensitivity in red blood cells - FR
Université Toulouse III
Unité de biologie Moléculaire, cellulaire et du développement

GERMANIA

Baden-Württemberg
FREIBURG

GERMANIA

Baden-Württemberg
FREIBURG

Finanziato da un ente associato a IRDiRCDBAGenCure: Lentiviral-mediated gene therapy for Diamond Blackfan Anemia: Preclinical Safety and Efficacy Studies -DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Pädiatrische Hämatologie und Onkologie

GERMANIA

Baden-Württemberg
FREIBURG

Finanziato da un ente associato a IRDiRCRIBOEUROPE: The European Ribosomopathy Consortium -DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Pädiatrische Hämatologie und Onkologie

GERMANIA

Baden-Württemberg
FREIBURG

TSP-Comp: Newly identified complement regulatory function of physiological proteins: Elucidating the mechanisms, physiological significance and potential for therapeutic applications
Zentrum für Kinder- und Jugendmedizin Freiburg
Sektion Kindernephrologie der Klinik für Allgemeine Kinder- und Jugendmedizin

GERMANIA

Baden-Württemberg
HEIDELBERG

Finanziato da un ente associato a IRDiRCADDRess: Translational research for patients with abnormal DNA Damage Response
Deutsches Krebsforschungszentrum
KITZ - Hopp-Kindertumorzentrum Heidelberg

GERMANIA

Baden-Württemberg
HEIDELBERG

Finanziato da un ente associato a IRDiRCADDRess: Translational research for patients with abnormal DNA Damage Response
Universitäts-Frauenklinik Heidelberg
Frauenheilkunde und Geburtshilfe

GERMANIA

Bayern
WÜRZBURG

Finanziato da un ente associato a IRDiRCADDRess: Translational research for patients with abnormal DNA Damage Response
Universität Würzburg - Biozentrum
Institut für Humangenetik

GERMANIA

Hessen
FRANKFURT AM MAIN

GERMANIA

Hessen
LANGEN

Preclinical Gene Therapy of Fanconi Anemia with Transposon-Based Approaches
Paul-Ehrlich-Institut
Abteilung Medizinische Biotechnologie

GERMANIA

Niedersachsen
HANNOVER

Finanziato da un ente associato a IRDiRCADDRess: Translational research for patients with abnormal DNA Damage Response
Medizinische Hochschule Hannover
Klinik für Pädiatrische Hämatologie und Onkologie

GERMANIA

Nordrhein-Westfalen
DÜSSELDORF

Finanziato da un ente associato a IRDiRCADDRess: Translational research for patients with abnormal DNA Damage Response
Universitätsklinikum Düsseldorf
Institut für Pathologie

GERMANIA

Nordrhein-Westfalen
KÖLN

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCProgressi nella diagnosi della talassemia e delle emoblobinopatie: sviluppo di tecniche innovative attraverso l'uso combinato di termogravimetria e chemiometria
ISS - Istituto Superiore di Sanità
Centro Nazionale per la Ricerca e la Valutazione preclinica e clinica dei Farmaci

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCRegistRare: the new National Platform dedicated to Rare Diseases Registries
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

ITALIA

LAZIO
ROMA

ITALIA

LOMBARDIA
MILANO

Finanziato da un ente associato a IRDiRCTC NER: Transcription stress Counteracted by Nutritional interventions of Exceptional importance for rare DNA Repair diseases - IT
IFOM - Istituto FIRC di Oncologia Molecolare
Firc Institute of Molecular Oncology (IFOM)

PORTOGALLO

SUL
LISBOA

Development and validation of vaso-occlusion early predictors in mendelian model of vascular disease
Instituto Nacional de Saúde Dr. Ricardo Jorge - Lisboa
Departamento de Genética

SPAGNA

Cataluña
BARCELONA

SPAGNA

Madrid
MADRID

Therapy of cancerous lesions of the oral mucosa of patients with Fanconi anemia using genetically corrected bioengineered autologous substitutes
CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
División de biomedicina epitelial

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCDBAGenCure: Lentiviral-mediated gene therapy for Diamond Blackfan anemia: Preclinical safety and efficacy studies - ES
CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
Terapias innovadoras en el sistema hematopoyético

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCDBAGenCure: Lentiviral-mediated gene therapy for Diamond Blackfan Anemia: Preclinical Safety and Efficacy Studies -DE
CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
Terapias innovadoras en el sistema hematopoyético

SPAGNA

Madrid
MADRID

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Hematología y Hemoterapia

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCDBAGenCure: Lentiviral-mediated gene therapy for Diamond Blackfan anemia: Preclinical safety and efficacy studies - ES
Hospital Materno Infantil Gregorio Marañón
Servicio de Oncología y Hematología Pediátricas y del Adolescente

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - ES
Hospital Universitario Fundación Jiménez Díaz

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCTherapeutics for rare and neglected diseases - science
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCPreclinical innovation
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 IRDiRCRepurposing an eu therapeutic for hemoglobinopathies
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCFanconi anemia:genotype-phenotype correlations
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCGenetic epidemiology of complex traits
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCHematopoietic stem cell biology
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCInsights into sickle cell trait and sickle cell disease
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCRed cell biology
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCFa ddr pathway in germline integrity
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCInsights into Sickle Cell Trait and Sickle Cell Disease
Institution: Information not provided - US

SVEZIA

Region Skåne
LUND

SVEZIA

Region Skåne
LUND

Finanziato da un ente associato a IRDiRCDEVDBA: Ontogeny as a critical determinant of DBA sensitivity in red blood cells - SE
Lunds Universitet
Department of Clinical Sciences (IKVL)

SVIZZERA

Suisse Alémanique
BERN

Thrombotic thrombocytopenic purpura - role of ADAMTS13 and long-term outcome
Universitätsspital Inselspital
Department of Hematology and Central Hematology Laboratory

SVIZZERA

Suisse Alémanique
ZURICH

Finanziato da un ente associato a IRDiRCFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - CH
ETH Zurich - Hönggerberg campus
Institute of Molecular Health Sciences

AUSTRIA

WIEN
ADDRESS: NOT PROVIDED - AT

AUSTRIA

WIEN
WIEN

Prospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie

FRANCIA

ILE-DE-FRANCE
LE KREMLIN-BICÊTRE

FRANCIA

ILE-DE-FRANCE
PARIS

Implementation of non-invasive prenatal diagnosis of rare and severe monogenic diseases
AP-HP.Centre - Université de Paris - Hôpital Cochin
Service de Médecine Génomique des Maladies de Système et d'Organe

FRANCIA

ILE-DE-FRANCE
PARIS

EGR2: Pregnancy and Medically Assisted Conception in Rare Diseases
AP-HP.Centre - Université de Paris - Hôpital Cochin
Unité fonctionnelle de Médecine interne et centre de référence maladies rares

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCAutoimmune cytopenias: genetics and pathophysiological mechanisms of pediatric Evans syndrome
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCIA

NOUVELLE AQUITAINE
BORDEAUX

Finanziato da un ente associato a IRDiRCSafety of CRISPR-Cas9 nuclease use
Université de Bordeaux ISPED
Centre de recherche INSERM U1219

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
NICE

Finanziato da un ente associato a IRDiRCRole of the Piezo1 / KCNN4 interaction in the pathophysiology of the red blood cell.
Université de Nice Sophia-Antipolis - Faculté des sciences
iBV - Institut de Biologie Valrose

GERMANIA

Baden-Württemberg
FREIBURG

Finanziato da un ente associato a IRDiRCGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
CCI am Universitätsklinikum Freiburg
Centrum für Chronische Immundefizienz

GERMANIA

Baden-Württemberg
HEIDELBERG

NGS2025 : Evaluation of the Expansion of Newborn screening by additional 28 target diseases
Zentrum für Kinder- und Jugendmedizin des Universitätsklinikums Heidelberg
Klinik für Kinderheilkunde I - Sektion für Neuropädiatrie und Stoffwechselmedizin

GERMANIA

Baden-Württemberg
TÜBINGEN

AWISPECS: The role of acquired von Willebrand syndrome in cardiosurgical interventions in infancy
Universitätsklinik für Kinder- und Jugendmedizin Tübingen
Abteilung für Kinderkardiologie, Pulmologie, Intensivmedizin

GERMANIA

Bayern
MÜNCHEN

Establishment of surface plasmon resonance (SPR) tests for improved diagnostics of autoimmune diseases
Klinikum rechts der Isar der Technischen Universität München
Arbeitsgruppe Biosensorik

GERMANIA

Niedersachsen
HANNOVER

Finanziato da un ente associato a IRDiRCGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Medizinische Hochschule Hannover
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie

GERMANIA

Niedersachsen
HANNOVER

Finanziato da un ente associato a IRDiRCGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Medizinische Hochschule Hannover
Klinik für Rheumatologie und Immunologie

GERMANIA

Nordrhein-Westfalen
KÖLN

REACT-2020: Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Universitätsklinikum Köln
Klinik II für Innere Medizin - Nephrologie, Rheumatologie, Diabetologie und allgemeine Innere Medizin

GERMANIA

Schleswig-Holstein
KIEL

Finanziato da un ente associato a IRDiRCGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Klinik für Innere Medizin I

IRLANDA

County Dublin
DUBLIN

The Low Von Willebrand factor Ireland Cohort of Kids (LoVIC-K) study
Royal College of Surgeons of Ireland
Molecular and Cellular Theraputics

ITALIA

LOMBARDIA
MILANO

ITALIA

LOMBARDIA
MILANO

Finanziato da un ente associato a IRDiRCSviluppo di vettori lentivirali per il trasferimento genetico nel fegato ed applicazioni alla terapia dell' Emofilia
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica

NORVEGIA

Nord-Norge
TROMSØ

Glycosylation of anti-HPA 1a antibodies and the potency for platelet clearence
UNN - Universitetssykehuset Nord-Norge
Diagnostisk klinikk

NORVEGIA

Nord-Norge
TROMSØ

Platelets and placenta - the new hotspot in fetal-maternal crosstalk
UNN - Universitetssykehuset Nord-Norge
Diagnostisk klinikk

NORVEGIA

Nord-Norge
TROMSØ

PORTOGALLO

SUL
LISBOA

Non-classical hereditary hemochromatosis and other rare genetic diseases associated with disorders in iron homeostasis
Instituto Nacional de Saúde Dr. Ricardo Jorge - Lisboa
Departamento de Genética

REGNO UNITO; GRAN BRETAGNA

Greater London
LONDON

Liver Disease in Pregnancy
King's College London
King's College London Headquarters

REGNO UNITO; GRAN BRETAGNA

Tyne & Wear
NEWCASTLE UPON TYNE

The Role of Membrane Attack Complex in Atypical Haemolytic Uraemic Syndrome
Institute of Cellular Medicine, Newcastle University, Medical School
Institute of Cellular Medicine

SPAGNA

Cataluña
BARCELONA

Analysis of the complement system as therapeutical target in severe preeclampsia and Hellp syndrome
Fundación Instituto de Investigación contra la Leucemia Josep Carreras
BET: Barcelona Endothelium Team

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCRole of platelet glycosylation on platelet function and immune response in immune thrombocytopenia patients
IdiPAZ - Instituto de Investigación Sanitaria del Hospital Universitario La Paz
Grupo de Coagulopatías y alteraciones de la hemostasia

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCQuantitative profiles of complement FH/FHR proteins as biomarkers of disease predisposition and evolution in primary and secondary haemolytic uraemic syndrome
IdiPAZ - Instituto de Investigación Sanitaria del Hospital Universitario La Paz
Laboratorio de Inmunopatología del Complemento

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCImproving diagnostic capacity and knowledge of pathogenesis of Primary Antiphospholipid syndrome through proteomics and transcriptomics
Instituto de Investigación Hospital 12 de Octubre
Fundación para la Investigación Biomédica del Hospital Universitario 12 de Octubre

SVIZZERA

Suisse Alémanique
ZÜRICH

The effect of cofactors and vitamins on homocysteine and methylmalonic acid metabolism in health and disease
Universitäts - Kinderspital Zürich - Eleonorenstiftung
Abteilung für Stoffwechselkrankheiten

AUSTRIA

WIEN
ADDRESS: NOT PROVIDED - AT

AHEAD: ADVATE Hemophilia A Outcome Database - AT
Institution: Information not provided - AT

AUSTRIA

WIEN
ADDRESS: NOT PROVIDED - AT

AUSTRIA

WIEN
ADDRESS: NOT PROVIDED - AT

CANADA

Ontario
KINGSTON

Finanziato da un ente associato a IRDiRCStudy of the factor VIII life cycle and its implications for factor VIII immunogenicity
Queen's University
Department of Pathology and Molecular Medicine

FRANCIA

GRAND-EST
ILLKIRCH-GRAFFENSTADEN

Search for Scott syndrom causative mutation(s)
Faculté de Pharmacie de Strasbourg
Laboratoire de Bioimagerie et Pathologies - LBP

FRANCIA

NOUVELLE AQUITAINE
PESSAC

Finanziato da un ente associato a IRDiRCChronic acquired thrombocytopenia of undetermined significance: pathophysiology and biomarker
CHU de Bordeaux-GH Sud - Hôpital Haut-Lévêque
Unité de Cardiopathies congénitales de l'enfant et de l'adolescent

GERMANIA

Bayern
MÜNCHEN

GEPHARD: German Pediatric Hemophilia Research Database
Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital
Hämophiliezentrum München - Pädiatrische Hämostaseologie

GERMANIA

Hessen
FRANKFURT AM MAIN

AHA_IIT: Long-term follow-up of therapy and outcome of acquired hemophilia A
Universitätsklinikum Frankfurt
Schwerpunkt Hämostaseologie/Hämophiliezentrum Frankfurt

GERMANIA

Nordrhein-Westfalen
BONN

Enzymological and structural biology investigation on the function of the vitamin K cycle
Universitätsklinikum Bonn (AöR)
Institut für Experimentelle Hämatologie und Transfusionsmedizin

ITALIA

UMBRIA
PERUGIA

NORVEGIA

Østlandet
OSLO

Factor (F) VII-deficiency: developing new therapeutic strategies to rescue FVII production and function
Oslo University Hospital, Ullevaal
Oslo University Hospital, Ullevål

REGNO UNITO; GRAN BRETAGNA

Greater Manchester
ADDRESS: NOT PROVIDED - UK

Finanziato da un ente associato a IRDiRCADVATE Hemophilia A Outcome Database (AHEAD) - UK
Institution: Information not provided - UK

SPAGNA

Cataluña
BARCELONA

PEAK Registry: Pyruvate Kinase Deficiency Global Longitudinal Registry - ES
Hospital Universitari Vall d'Hebron
Servicio de Hematología y Hemoterapia

SPAGNA

Comunidad Valenciana
VALENCIA

Personalized Prophylactic Treatment With Advate® in Severe or Moderate Haemophilia A Patients
Hospital Universitario y Politécnico La Fe
Servicio de Farmacia, Área Clínica del Medicamento

SPAGNA

Madrid
ALCORCÓN

Finanziato da un ente associato a IRDiRCDeciphering the role of factor-H related proteins in complement-related kidney diseases
Hospital Universitario Fundación Alcorcón
Servicio de Nefrología

SPAGNA

Madrid
MADRID

Preclinical evaluation of the efficacy and safety of a new lentiviral vector for the gene therapy of Glazmann Thrombasthenia
CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
Terapias innovadoras en el sistema hematopoyético

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCDeciphering the role of factor-H related proteins in complement-related kidney diseases
Instituto de Investigación Hospital 12 de Octubre
Grupo de investigación en Nefrología Clínica

CANADA

Ontario
OTTAWA

Finanziato da un ente associato a IRDiRCNovel therapeutic approaches to target GNE Myopathy
Children's Hospital of Eastern Ontario
Division of Neurology

FRANCIA

AUVERGNE-RHONE-ALPES
PRAGUE

Finanziato da un ente associato a IRDiRCRole on Nonmuscle Myosin II in membrane trafficking and organ function
Faculty of Medicine and University Hospital Motol
Department of Pediatrics

FRANCIA

GRAND-EST
STRASBOURG

Finanziato da un ente associato a IRDiRCBernard-Soulier Syndrome and MYH9 associated diseases: study of blood platelets anomalies and of the thrombopoïesis, correction by genetic therapy
Établissement Français du Sang - Grand Est
UR U311 - Biologie et pharmacologie de l'hémostase et de la thrombose

GERMANIA

Sachsen-Anhalt
HALLE (SAALE)

Finanziato da un ente associato a IRDiRCProDGNE: Novel therapeutic approaches to target GNE Myopathy -DE
Institut für Physiologische Chemie der Medizinische Fakultät
Institut für Physiologische Chemie

ISRAELE

ISRAEL
ADDRESS: NOT PROVIDED - IL

Finanziato da un ente associato a IRDiRCZebrafish as a model system for GNE Myopathy
Institution: Information not provided - IL

ISRAELE

ISRAEL
JERUSALEM

Finanziato da un ente associato a IRDiRCCellular Models to explore GNE functions in muscle
The Hebrew University of Jerusalem
Faculty of Medicine

ITALIA

SARDEGNA
CAGLIARI

Finanziato da un ente associato a IRDiRCProDGNE: Nuovi approcci terapeutici indirizzati alla miopatia GNE - IT
Università degli Studi di Cagliari
Dipartimento di Scienze della Vita e dell'Ambiente

SPAGNA

Madrid
MADRID

Finanziato da un ente associato a IRDiRCStudy of the procoagulant effect of emicizumab and of its combination with factor concentrates for the treatment of bleeds in patients with severe haemophilia A
IdiPAZ - Instituto de Investigación Sanitaria del Hospital Universitario La Paz
Grupo de Coagulopatías y alteraciones de la hemostasia

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCCell biology of metabolic disorders
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 IRDiRCClinical and translational studies of RUNX1 and FPD
Institution: Information not provided - US

FRANCIA

GRAND-EST
STRASBOURG

Characterisation of mutations responsible for Bernard-Soulier syndrome (GP1BA, BP1BB and GP9 genes)
Établissement Français du Sang - Grand Est
UR U311 - Biologie et pharmacologie de l'hémostase et de la thrombose

ITALIA

LOMBARDIA
MILANO

Production of vectors for gene therapy (Wiskott-Aldrich Syndrome and Metachromatic Leukodystrophy)
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica

ITALIA

LOMBARDIA
MILANO

Finanziato da un ente associato a IRDiRCSindrome di Wiskott-Aldrich: caratterizzazione dei difetti immunologici e studi preclinici di terapia genica
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica

STATI UNITI

Maryland
BETHESDA

Finanziato da un ente associato a IRDiRCNovel Treatment for Hermansky-Pudlak Syndrome Pulmonary Fibrosis
National Center for Advancing Translational Sciences (NCATS-NIH)
Therapeutic Development Branch

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCGene therapy approaches for primary immunodeficiencies
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMolecular and clinical studies of primary immunodeficiencies
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 IRDiRCClinical research of oral connective tissue program
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCProtein trafficking in the endosomal-lysosomal system
Institution: Information not provided - US

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCRole of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

REGNO UNITO; GRAN BRETAGNA

Cambridgeshire
CAMBRIDGE

Progetti di ricerca multicentrici