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Projetos de investigação

Inherited bone marrow failure syndromes: from genomic discoveries to biology Hospital for Sick Children, Research Institute

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

Mutational spectra and clinical manifestation in patients with congenital haemophilia Institut für Humangenetik der Universitätsmedizin Greifswald Institut für Humangenetik

Diagnosis and treatment of acquired hemostatic disorders due to auto-antibodies CHU Paris-Sud - Hôpital de Bicêtre Service d'Hématologie

Input of a multidisciplinary management approach in announcing the diagnosis for young children with serious constitutional hemorrhagic disease and their families CHU de Marseille - Hôpital de la Timone Service d'Hématologie Immunologie Oncologie Pédiatrique

Clinical research on the pathophysiology, diagnosis and management of idiopathic thrombosis in neonates, infants and children Kyushu University Graduate School of Medical Sciences Department of Pediatrics

Bacterial virulence factors, innate immunity and prothrombotic mechanisms in renal disease Skånes Universitetssjukhus Department of Clinical Sciences, Division of Pediatrics

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

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

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

Pheno- and genotypes of JAK2 mutated patients with and without thromboembolic events. A project of the East German Study Group (OSHO) Universitätsklinikum Halle (Saale) Krukenberg-Krebszentrum Halle (KKH)

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

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

Identification of new factors involved in the onset, progression, and complications of Philadelphia-negative chronic myeloproliferative neoplasia IMIB - Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca Grupo de Hematología y Oncología Médica Clínico-Experimental

Immunological characterisation and modulation of the immune response anti-factor VIII in hemophilia A, and anti-factor IX in hemophilia B CHU Paris-Sud - Hôpital de Bicêtre Service d'Hématologie

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

Irish Personalised Approach to the Treatment of Haemophilia (iPATH) Children's Health Ireland @ Crumlin Haematology Department

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

Irish Personalised Approach to the Treatment of Haemophilia (iPATH) Science Foundation Ireland

Irish Personalised Approach to the Treatment of Haemophilia (iPATH) St James's Hospital National Coagulation Centre

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

Development 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

Development of a predictive method for bleeding complication associated with intractable cardiovascular diseases with high shear-stress Institute of Development and Aging and Cancer,Tohoku University Department of Molecular and Cellular Biology

Development of management for acquired von Wilebrand syndrome complicated with mechanical circulatory support Tohoku University Graduate School of Medicine Department of Cardiovascular Surgery

Role of regulatory elements in the genetic determination of VWF level in Von Willebrand disease and healthy individuals University of Cambridge Departmet of Haematology

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

Molecular mechanisms and clinical consequences of variable fibrinogen levels Centre Médical Universitaire - CMU Département de médecine génétique et développement

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

MOTIVATE: MOdern Treatment of Inhibitor-PositiVe PATiEnts With Hemophilia A - An International Observational Study Hämophilie-Zentrum Rhein Main GmbH

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

Enzymological and structural biology investigation on the function of the vitamin K cycle Deutsche Forschungsgemeinschaft

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

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

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

HEM-POWR: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A - AT Institution: Information not provided - AT

A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment - AT Institution: Information not provided - AT

A Prospective, Non-Interventional Study to investigate the Effectiveness of AFSTYLA® in Patients with Hemophilia A - AT Institution: Information not provided - AT

Study of the factor VIII life cycle and its implications for factor VIII immunogenicity Queen's University Department of Pathology and Molecular Medicine

Differential Diagnostic of ITP and MDS: a Prospective Study by Next-Generation Flow Cytometry and Cytomorphological Approaches Hospital Universitario de Burgos Servicio de Hematología y Hemoterapia

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

Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using an Online Medical Application Based on a Published Population Model and a Bayesian Algorithm - ES Hospital Universitario La Paz Servicio de Hematología

Efficacy of a Prophylactic Treatment Using Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Adult Patients With Hemophilia. Observational Multicenter Study - ES Universidad Europea de Madrid. Facultad de Ciencias de la Salud Departamento de Fisioterapia

Molecular pathology of antithrombin deficiency: development of new diagnostic methods and clinical implications IMIB - Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca Grupo de Hematología y Oncología Médica Clínico-Experimental

HEM-POWR: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A - FR Institution: Information not provided - FR

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

Predictive response factors to the treatment of Rituximab on B and T lymphocytes sub-populations in the immunological thrombocytopenic purpura in adults (RITUX-PTI study) CHU Henri Mondor Service de Médecine interne

Constitutive hematologic diseases: clinical and therapeutic research, molecular biology CHU Paris-GH St-Louis Lariboisière F.Widal - Hôpital Saint-Louis Service d'hématologie pédiatrique

Dissecting dendritic cell subsets as key to installing tolerance to FVIII in hemophilia A Università degli Studi di Perugia Dipartimento di Medicina Sperimentale

Establishment of diagnostic system, registry and sample banking in congenital thrombocytopenia National Center for Child Health and Development Research Institute

ADVATE Hemophilia A Outcome Database (AHEAD) - UK Institution: Information not provided - UK

A-SURE: 24-month Prospective, Multicentre, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products in the Prophylactic Treatment of Patients With Haemophilia A - AT Institution: Information not provided - AT

DYNAMO: The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B - AT Institution: Information not provided - AT

Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia - AT Institution: Information not provided - AT

A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults With Hemophilia - AT Allgemeines Krankenhaus der Stadt Wien Klinische Abteilung für Hämatologie und Hämostaseologie

Canadian Hemophilia dose Escalation Primary Prophylaxis (CHPS) Observational Extension Study Hospital for Sick Children, Research Institute

Study 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

Cell biology of metabolic disorders Institution: Information not provided - US

Functional and translational studies of runx1 and cbfb in hematopoiesis Institution: Information not provided - US

Role on Nonmuscle Myosin II in membrane trafficking and organ function Faculty of Medicine and University Hospital Motol Department of Pediatrics

Bernard-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

Zebrafish as a model system for GNE Myopathy Institution: Information not provided - IL

A silk-based bone marrow model to predict individual response to old and new drugs for increasing platelet count in inherited thrombocytopenias Fondazione IRCCS Policlinico San Matteo Clinica Medica III

Hereditary Inclusion Body Myopathy-Patient Monitoring Program (HIBM-PMP): A Registry and Prospective Observational Natural History Study to Assess HIBM Disease Newcastle University John Walton Muscular Dystrophy Research Centre, Institute for Genetic Medicine

In vitro modeling of primary immunodeficiencies with defects in NK cells Instituto de Investigación sanitaria Biocruces Bizkaia Grupo de investigación en terapia celular, células madre y tejidos

Gene therapy approaches for primary immunodeficiencies Institution: Information not provided - US

Molecular and clinical studies of primary immunodeficiencies Institution: Information not provided - US

National ophthalmic disease genotyping and phenotyping network - eyegene Institution: Information not provided - US

Clinical research of oral connective tissue program Institution: Information not provided - US

Protein trafficking in the endosomal-lysosomal system Institution: Information not provided - US

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

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

Wiskott-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

The Fundamentals of Phagocytosis: Integrating Theoretical Models and Experiments University of Exeter, Physics building Department of Physics and Astronomy

Unraveling the mechanisms for development of primary immunodeficiency using experimental models (Wiskott-Aldrich syndrome and X-linked severe congenital neutropenia) Karolinska Institutet - Huddinge Division of Clinical Chemistry

Role of the Wiskott-Aldrich Syndrome Protein (WASp) in the Differentiation and Regulation of the Immune System Centre Hospitalier Universitaire Vaudois CHUV Service d'immunologie et allergie

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

Inherited bone marrow failure syndromes: from genomic discoveries to biology
Hospital for Sick Children, Research Institute

RBDD: Rare Bleeding Disorders Database network (coordination)
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Laboratorio di Patologie Molecolari Applicate alla Clinica

Mutational spectra and clinical manifestation in patients with congenital haemophilia
Institut für Humangenetik der Universitätsmedizin Greifswald

Institut für Humangenetik

Diagnosis and treatment of acquired hemostatic disorders due to auto-antibodies
CHU Paris-Sud - Hôpital de Bicêtre

Service d'Hématologie

Input of a multidisciplinary management approach in announcing the diagnosis for young children with serious constitutional hemorrhagic disease and their families
CHU de Marseille - Hôpital de la Timone

Service d'Hématologie Immunologie Oncologie Pédiatrique

Clinical research on the pathophysiology, diagnosis and management of idiopathic thrombosis in neonates, infants and children
Kyushu University Graduate School of Medical Sciences

Department of Pediatrics

Bacterial virulence factors, innate immunity and prothrombotic mechanisms in renal disease
Skånes Universitetssjukhus

Department of Clinical Sciences, Division of Pediatrics

Thrombotic thrombocytopenic purpura - role of ADAMTS13 and long-term outcome
University Hospital Inselspital

Department of Hematology and Central Hematology Laboratory

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

Abteilung für Kinderkardiologie, Pulmologie, Intensivmedizin

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

Arbeitsgruppe Biosensorik

Pheno- and genotypes of JAK2 mutated patients with and without thromboembolic events. A project of the East German Study Group (OSHO)
Universitätsklinikum Halle (Saale)

Krukenberg-Krebszentrum Halle (KKH)

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

Klinische Abteilung für Hämatologie

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

Identification of new factors involved in the onset, progression, and complications of Philadelphia-negative chronic myeloproliferative neoplasia
IMIB - Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca

Grupo de Hematología y Oncología Médica Clínico-Experimental

Immunological characterisation and modulation of the immune response anti-factor VIII in hemophilia A, and anti-factor IX in hemophilia B
CHU Paris-Sud - Hôpital de Bicêtre

Service d'Hématologie

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

Irish Personalised Approach to the Treatment of Haemophilia (iPATH)
Children's Health Ireland @ Crumlin

Haematology Department

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

Molecular and Cellular Theraputics

Irish Personalised Approach to the Treatment of Haemophilia (iPATH)
Science Foundation Ireland

Irish Personalised Approach to the Treatment of Haemophilia (iPATH)
St James's Hospital

National Coagulation Centre

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

Development 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

Development of a predictive method for bleeding complication associated with intractable cardiovascular diseases with high shear-stress
Institute of Development and Aging and Cancer,Tohoku University

Department of Molecular and Cellular Biology

Development of management for acquired von Wilebrand syndrome complicated with mechanical circulatory support
Tohoku University Graduate School of Medicine

Department of Cardiovascular Surgery

Role of regulatory elements in the genetic determination of VWF level in Von Willebrand disease and healthy individuals
University of Cambridge

Departmet of Haematology

Liver Disease in Pregnancy
King's College London

King's College London Headquarters

Molecular mechanisms and clinical consequences of variable fibrinogen levels
Centre Médical Universitaire - CMU

Département de médecine génétique et développement

GAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
CCI am Universitätsklinikum Freiburg

Centrum für Chronische Immundefizienz

MOTIVATE: MOdern Treatment of Inhibitor-PositiVe PATiEnts With Hemophilia A - An International Observational Study
Hämophilie-Zentrum Rhein Main GmbH

GAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Medizinische Hochschule Hannover

Klinik für Rheumatologie und Immunologie

Enzymological and structural biology investigation on the function of the vitamin K cycle
Deutsche Forschungsgemeinschaft

Enzymological and structural biology investigation on the function of the vitamin K cycle
Universitätsklinikum Bonn (AöR)

Institut für Experimentelle Hämatologie und Transfusionsmedizin

GAIN: German network for research and therapy optimization of patients with multi-organ autoimmune diseases
Universitätsklinikum Schleswig-Holstein - Campus Kiel

Klinik für Innere Medizin I

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

HEM-POWR: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A - AT
Institution: Information not provided - AT

A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment - AT
Institution: Information not provided - AT

A Prospective, Non-Interventional Study to investigate the Effectiveness of AFSTYLA® in Patients with Hemophilia A - AT
Institution: Information not provided - AT

Study of the factor VIII life cycle and its implications for factor VIII immunogenicity
Queen's University

Department of Pathology and Molecular Medicine

Differential Diagnostic of ITP and MDS: a Prospective Study by Next-Generation Flow Cytometry and Cytomorphological Approaches
Hospital Universitario de Burgos

Servicio de Hematología y Hemoterapia

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

Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using an Online Medical Application Based on a Published Population Model and a Bayesian Algorithm - ES
Hospital Universitario La Paz

Servicio de Hematología

Efficacy of a Prophylactic Treatment Using Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Adult Patients With Hemophilia. Observational Multicenter Study - ES
Universidad Europea de Madrid. Facultad de Ciencias de la Salud

Departamento de Fisioterapia

Molecular pathology of antithrombin deficiency: development of new diagnostic methods and clinical implications
IMIB - Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca

Grupo de Hematología y Oncología Médica Clínico-Experimental

HEM-POWR: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A - FR
Institution: Information not provided - FR

Search for Scott syndrom causative mutation(s)
Faculté de Pharmacie de Strasbourg

Laboratoire de Bioimagerie et Pathologies - LBP

Predictive response factors to the treatment of Rituximab on B and T lymphocytes sub-populations in the immunological thrombocytopenic purpura in adults (RITUX-PTI study)
CHU Henri Mondor

Service de Médecine interne

Constitutive hematologic diseases: clinical and therapeutic research, molecular biology
CHU Paris-GH St-Louis Lariboisière F.Widal - Hôpital Saint-Louis

Service d'hématologie pédiatrique

Dissecting dendritic cell subsets as key to installing tolerance to FVIII in hemophilia A
Università degli Studi di Perugia

Dipartimento di Medicina Sperimentale

Establishment of diagnostic system, registry and sample banking in congenital thrombocytopenia
National Center for Child Health and Development

Research Institute

ADVATE Hemophilia A Outcome Database (AHEAD) - UK
Institution: Information not provided - UK

A-SURE: 24-month Prospective, Multicentre, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products in the Prophylactic Treatment of Patients With Haemophilia A - AT
Institution: Information not provided - AT

DYNAMO: The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B - AT
Institution: Information not provided - AT

Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia - AT
Institution: Information not provided - AT

A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults With Hemophilia - AT
Allgemeines Krankenhaus der Stadt Wien

Klinische Abteilung für Hämatologie und Hämostaseologie

Canadian Hemophilia dose Escalation Primary Prophylaxis (CHPS) Observational Extension Study
Hospital for Sick Children, Research Institute

Study 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

Cell biology of metabolic disorders
Institution: Information not provided - US

Functional and translational studies of runx1 and cbfb in hematopoiesis
Institution: Information not provided - US

Role on Nonmuscle Myosin II in membrane trafficking and organ function
Faculty of Medicine and University Hospital Motol

Department of Pediatrics

Bernard-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

Zebrafish as a model system for GNE Myopathy
Institution: Information not provided - IL

A silk-based bone marrow model to predict individual response to old and new drugs for increasing platelet count in inherited thrombocytopenias
Fondazione IRCCS Policlinico San Matteo

Clinica Medica III

Hereditary Inclusion Body Myopathy-Patient Monitoring Program (HIBM-PMP): A Registry and Prospective Observational Natural History Study to Assess HIBM Disease
Newcastle University

John Walton Muscular Dystrophy Research Centre, Institute for Genetic Medicine

In vitro modeling of primary immunodeficiencies with defects in NK cells
Instituto de Investigación sanitaria Biocruces Bizkaia

Grupo de investigación en terapia celular, células madre y tejidos

Gene therapy approaches for primary immunodeficiencies
Institution: Information not provided - US

Molecular and clinical studies of primary immunodeficiencies
Institution: Information not provided - US

National ophthalmic disease genotyping and phenotyping network - eyegene
Institution: Information not provided - US

Clinical research of oral connective tissue program
Institution: Information not provided - US

Protein trafficking in the endosomal-lysosomal system
Institution: Information not provided - US

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

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

Wiskott-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

The Fundamentals of Phagocytosis: Integrating Theoretical Models and Experiments
University of Exeter, Physics building

Department of Physics and Astronomy

Unraveling the mechanisms for development of primary immunodeficiency using experimental models (Wiskott-Aldrich syndrome and X-linked severe congenital neutropenia)
Karolinska Institutet - Huddinge

Division of Clinical Chemistry

Role of the Wiskott-Aldrich Syndrome Protein (WASp) in the Differentiation and Regulation of the Immune System
Centre Hospitalier Universitaire Vaudois CHUV

Service d'immunologie et allergie

Role of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità

Centro Nazionale Malattie Rare

Understanding granule disorders of myeloid cells by unravelling the interactome and function of the Nbeal2 protein
University of Cambridge

Departmet of Haematology

RBDD: Rare Bleeding Disorders Database network

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Laboratorio di Patologie Molecolari Applicate alla Clinica

PERSIST: Persisting transgenesis (TERMINATED)

Istituto San Raffaele Telethon per la Terapia Genica - TIGET

Istituto San Raffaele Telethon per la Terapia Genica

PROFNAIT: Development of a prophylactic treatment for the prevention of fetal/neonatal alloimmune thrombocytopenia (FNAIT)

Prophylix Pharma AS