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Research projects

CANADA

Ontario
TORONTO

AUSTRIA

STEIERMARK
GRAZ

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

FRANCE

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

FRANCE

NOUVELLE AQUITAINE
BORDEAUX

GERMANY

Berlin
ADDRESS: NOT PROVIDED - DE

GERMANY

Sachsen-Anhalt
HALLE (SAALE)

ITALY

EMILIA ROMAGNA
MELDOLA

Integrated Multiomics and Multilevel Characterization of Haematological Disorders and Malignancies
IRST - Istituto Scientifico Romagnolo per lo studio e la cura dei Tumori
IRCCS Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori

ITALY

LOMBARDIA
MILANO

RBDD: Rare Bleeding Disorders Database network (coordination)
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Laboratorio di Patologie Molecolari Applicate alla Clinica

SWITZERLAND

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

FRANCE

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

PORTUGAL

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

Funded by an IRDiRC memberRIBOEUROPE: 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

BELGIUM

HAINAUT
GOSSELIES

Funded by an IRDiRC memberRIBOEUROPE: The European Ribosomopathy Consortium - BE
Biopark campus
The 'RNA Metabolism' Lab - Université Libre de Bruxelles (ULB)

BELGIUM

HAINAUT
GOSSELIES

Funded by an IRDiRC memberDBAGenCure: 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

Funded by an IRDiRC memberPathobiology and robosomal insufficiency in Shwachman-Diamond syndrome
Hospital for Sick Children, Research Institute

FINLAND

Finland
HELSINKI

Funded by an IRDiRC memberGenomic editing for treating hemoglobin diseases
University of Helsinki
Stem Cells and Metabolism Research Program

FRANCE

GRAND-EST
STRASBOURG

FRANCE

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

FRANCE

ILE-DE-FRANCE
PARIS

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

FRANCE

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

FRANCE

ILE-DE-FRANCE
PARIS

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

FRANCE

ILE-DE-FRANCE
PARIS

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberRIBOEUROPE: 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)

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - FR
GHU AP-HP Nord. Université de Paris - Hôpital Saint-Louis
Laboratoire d'hématologie

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberCRISPR/Cas9 mediated induction of fetal hemoglobin synthesis for the treatment of beta-hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberDevelopment of Innovative Therapeutic Strategies for Beta-Hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCE

ILE-DE-FRANCE
PARIS

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberDevelopment of a universal prime editing approach to beta-hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCE

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

FRANCE

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

FRANCE

OCCITANIE
TOULOUSE

Funded by an IRDiRC memberRIBODBA: Blackfan-Diamond anemia: molecular mechanisms of a ribosomal disease
Université Paul Sabatier
Laboratoire de Biologie Moléculaire Eucaryote - UMR 5099

FRANCE

OCCITANIE
TOULOUSE

Funded by an IRDiRC memberDEVDBA: Ontogeny as a critical determinant of DBA sensitivity in red blood cells - FR
Université Toulouse III
Unité de biologie Moléculaire, cellulaire et du développement

GERMANY

Baden-Württemberg
FREIBURG

GERMANY

Baden-Württemberg
FREIBURG

Funded by an IRDiRC memberDBAGenCure: 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

GERMANY

Baden-Württemberg
FREIBURG

Funded by an IRDiRC memberRIBOEUROPE: The European Ribosomopathy Consortium -DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Pädiatrische Hämatologie und Onkologie

GERMANY

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

GERMANY

Baden-Württemberg
HEIDELBERG

Funded by an IRDiRC memberADDRess: Translational research for patients with abnormal DNA Damage Response
Deutsches Krebsforschungszentrum
KITZ - Hopp-Kindertumorzentrum Heidelberg

GERMANY

Baden-Württemberg
HEIDELBERG

Funded by an IRDiRC memberADDRess: Translational research for patients with abnormal DNA Damage Response
Universitäts-Frauenklinik Heidelberg
Frauenheilkunde und Geburtshilfe

GERMANY

Bayern
WÜRZBURG

Funded by an IRDiRC memberADDRess: Translational research for patients with abnormal DNA Damage Response
Universität Würzburg - Biozentrum
Institut für Humangenetik

GERMANY

Hessen
FRANKFURT AM MAIN

GERMANY

Hessen
LANGEN

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

GERMANY

Niedersachsen
HANNOVER

Funded by an IRDiRC memberADDRess: Translational research for patients with abnormal DNA Damage Response
Medizinische Hochschule Hannover
Klinik für Pädiatrische Hämatologie und Onkologie

GERMANY

Nordrhein-Westfalen
DÜSSELDORF

Funded by an IRDiRC memberADDRess: Translational research for patients with abnormal DNA Damage Response
Universitätsklinikum Düsseldorf
Institut für Pathologie

GERMANY

Nordrhein-Westfalen
KÖLN

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberAdvances in diagnosis of thalassemia and haemoglobinopathies: development of innovative techniques by thermogravimetry coupled with chemometrics
ISS - Istituto Superiore di Sanità
Centro Nazionale per la Ricerca e la Valutazione preclinica e clinica dei Farmaci

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberRegistRare: the new National Platform dedicated to Rare Diseases Registries
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberImproving quality standards in Italian laboratories performing genetic testing for rare diseases
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberTC 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)

KOREA, REPUBLIC OF

KOREA, REPUBLIC OF
SEOUL

PORTUGAL

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

SPAIN

Cataluña
BARCELONA

SPAIN

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

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberDBAGenCure: 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

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberDBAGenCure: 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

SPAIN

Madrid
MADRID

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Hematología y Hemoterapia

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberDBAGenCure: 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

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - ES
Hospital Universitario Fundación Jiménez Díaz

SWEDEN

Region Skåne
LUND

SWEDEN

Region Skåne
LUND

Funded by an IRDiRC memberDEVDBA: Ontogeny as a critical determinant of DBA sensitivity in red blood cells - SE
Lunds Universitet
Department of Clinical Sciences (IKVL)

SWITZERLAND

Suisse Alémanique
BERN

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

SWITZERLAND

Suisse Alémanique
ZURICH

Funded by an IRDiRC memberFANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - CH
ETH Zurich - Hönggerberg campus
Institute of Molecular Health Sciences

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberTherapeutics for rare and neglected diseases - science
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberPreclinical innovation
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRepurposing an eu therapeutic for hemoglobinopathies
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberFanconi anemia:genotype-phenotype correlations
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberGenetic epidemiology of complex traits
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberHematopoietic stem cell biology
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberInsights into sickle cell trait and sickle cell disease
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRed cell biology
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberFa ddr pathway in germline integrity
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberInsights into Sickle Cell Trait and Sickle Cell Disease
Institution: Information not provided - US

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

FRANCE

ILE-DE-FRANCE
LE KREMLIN-BICÊTRE

FRANCE

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

FRANCE

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

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberAutoimmune cytopenias: genetics and pathophysiological mechanisms of pediatric Evans syndrome
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163

FRANCE

NOUVELLE AQUITAINE
BORDEAUX

Funded by an IRDiRC memberSafety of CRISPR-Cas9 nuclease use
Université de Bordeaux ISPED
Centre de recherche INSERM U1219

FRANCE

PROVENCE-ALPES-COTE D'AZUR
NICE

Funded by an IRDiRC memberRole 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

GERMANY

Baden-Württemberg
FREIBURG

Funded by an IRDiRC memberGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
CCI am Universitätsklinikum Freiburg
Centrum für Chronische Immundefizienz

GERMANY

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

GERMANY

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

GERMANY

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

GERMANY

Niedersachsen
HANNOVER

Funded by an IRDiRC memberGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Medizinische Hochschule Hannover
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie

GERMANY

Niedersachsen
HANNOVER

Funded by an IRDiRC memberGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Medizinische Hochschule Hannover
Klinik für Rheumatologie und Immunologie

GERMANY

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

GERMANY

Schleswig-Holstein
KIEL

Funded by an IRDiRC memberGAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Klinik für Innere Medizin I

IRELAND

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

ITALY

LOMBARDIA
MILANO

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberDevelopment of lentiviral vectors for liver gene transfer and application to hemophilia gene therapy
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica

NORWAY

Nord-Norge
TROMSØ

Undersøkelse av karbohydrater bundet til antistoff rettet mot blodplateantigen
UNN - Universitetssykehuset Nord-Norge
Diagnostisk klinikk

NORWAY

Nord-Norge
TROMSØ

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

NORWAY

Nord-Norge
TROMSØ

PORTUGAL

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

SPAIN

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

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberRole 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

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberQuantitative 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

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberImproving 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

SWITZERLAND

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

UNITED KINGDOM

Greater London
LONDON

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

UNITED KINGDOM

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

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

Funded by an IRDiRC memberStudy of the factor VIII life cycle and its implications for factor VIII immunogenicity
Queen's University
Department of Pathology and Molecular Medicine

FRANCE

GRAND-EST
ILLKIRCH-GRAFFENSTADEN

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

FRANCE

NOUVELLE AQUITAINE
PESSAC

Funded by an IRDiRC memberChronic 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

GERMANY

Bayern
MÜNCHEN

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

GERMANY

Hessen
FRANKFURT AM MAIN

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

GERMANY

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

ITALY

UMBRIA
PERUGIA

Funded by an IRDiRC memberDissecting dendritic cell subsets as key to installing tolerance to FVIII in hemophilia A
Università degli Studi di Perugia
Dipartimento di Medicina Sperimentale

NORWAY

Østlandet
OSLO

Skandinavisk populasjonsbasert-PK studie hos pasienter med hemofili
Oslo University Hospital, Rikshospitalet
Pediatrisk forskningsinstitutt

NORWAY

Østlandet
OSLO

Faktor VII-mangel (CRISPR): Forsøk på å reparere genskade hos pasienter med faktor VII-mangel
Oslo University Hospital, Ullevaal
Oslo University Hospital, Ullevål

SPAIN

Cataluña
BARCELONA

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

SPAIN

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

SPAIN

Madrid
ALCORCÓN

Funded by an IRDiRC memberDeciphering the role of factor-H related proteins in complement-related kidney diseases
Hospital Universitario Fundación Alcorcón
Servicio de Nefrología

SPAIN

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

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberDeciphering 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

UNITED KINGDOM

Greater Manchester
ADDRESS: NOT PROVIDED - UK

Funded by an IRDiRC memberADVATE Hemophilia A Outcome Database (AHEAD) - UK
Institution: Information not provided - UK

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberNovel therapeutic approaches to target GNE Myopathy
Children's Hospital of Eastern Ontario
Division of Neurology

CANADA

Ontario
TORONTO

FRANCE

AUVERGNE-RHONE-ALPES
PRAGUE

Funded by an IRDiRC memberRole on Nonmuscle Myosin II in membrane trafficking and organ function
Faculty of Medicine and University Hospital Motol
Department of Pediatrics

FRANCE

GRAND-EST
STRASBOURG

Funded by an IRDiRC memberBernard-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

GERMANY

Sachsen-Anhalt
HALLE (SAALE)

Funded by an IRDiRC memberProDGNE: Novel therapeutic approaches to target GNE Myopathy -DE
Institut für Physiologische Chemie der Medizinische Fakultät
Institut für Physiologische Chemie

ISRAEL

ISRAEL
ADDRESS: NOT PROVIDED - IL

Funded by an IRDiRC memberZebrafish as a model system for GNE Myopathy
Institution: Information not provided - IL

ISRAEL

ISRAEL
JERUSALEM

Funded by an IRDiRC memberCellular Models to explore GNE functions in muscle
The Hebrew University of Jerusalem
Faculty of Medicine

ITALY

SARDEGNA
CAGLIARI

Funded by an IRDiRC memberProDGNE: Novel therapeutic approaches to target GNE Myopathy - IT
Università degli Studi di Cagliari
Dipartimento di Scienze della Vita e dell'Ambiente

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberStudy 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

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberCell biology of metabolic disorders
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberClinical and translational studies of RUNX1 and FPD
Institution: Information not provided - US

FRANCE

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

ITALY

LOMBARDIA
MILANO

Produzione vettori per la terapia genica (Sindrome di Wiskott-Aldrich e Leucodistrofia Metacromatica)
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberWiskott-Aldrich syndrome: characterization of immune system defects and gene therapy preclinical studies
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica

UNITED STATES

Maryland
BETHESDA

Funded by an IRDiRC memberNovel Treatment for Hermansky-Pudlak Syndrome Pulmonary Fibrosis
National Center for Advancing Translational Sciences (NCATS-NIH)
Therapeutic Development Branch

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberGene therapy approaches for primary immunodeficiencies
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMolecular and clinical studies of primary immunodeficiencies
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberClinical research of oral connective tissue program
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberProtein trafficking in the endosomal-lysosomal system
Institution: Information not provided - US

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberRole of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

UNITED KINGDOM

Cambridgeshire
CAMBRIDGE

Multicentric Research projects