Orphanet: Ricerca per malattia/gene
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Finanziato da un ente associato a IRDiRC = Membro di una ERN =

Progetti di ricerca

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

GERMANIA

Baden-Württemberg
FREIBURG

Finanziato da un ente associato a IRDiRCApoptoMDS: Hematopoietic stem cell Apoptosis in bone marrow failure and MyeloDysplastic Syndromes: Friend or foe? - DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Pädiatrische Hämatologie und Onkologie

SVIZZERA

Suisse Alémanique
BASEL

AUSTRIA

STEIERMARK
GRAZ

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

CANADA

Ontario
TORONTO

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

CANADA

Ontario
TORONTO

COREA DEL SUD

KOREA, REPUBLIC OF
SEOUL

FRANCIA

ILE-DE-FRANCE
PARIS

Physiopathology of human myelofibrosis with myeloid metaplasia
CHU Paris Est - Hôpital Saint-Antoine
UF de cytogénétique onco-hématologique

FRANCIA

ILE-DE-FRANCE
PARIS

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

FRANCIA

OCCITANIE
TOULOUSE

RIBODBA: Blackfan-Diamond anemia: molecular mechanisms of a ribosomal disease
Université Paul Sabatier
Laboratoire de Biologie Moléculaire Eucaryote - UMR 5099

GERMANIA

Baden-Württemberg
FREIBURG

GERMANIA

Bayern
WÜRZBURG

GERMANIA

Hessen
LANGEN

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

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

SPAGNA

Andalucía
MÁLAGA

Systems medicine approaches for the identification of new therapeutic targets in rare genetic diseases
Universidad de Málaga. Facultad de Ciencias
Departamento de Biología Molecular y Bioquímica

SPAGNA

Andalucía
SEVILLA

Mathematical models of disease mechanisms to reformulate drugs for rare diseases
Fundación Progreso y Salud
Área de bioinformática clínica

SPAGNA

Madrid
MADRID

Preclinical studies to demonstrate the efficacy and the safety of an ex vivo gene therapy approach in Diamond Blackfan Anemia with lentiviral vectors
CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
Terapias innovadoras en el sistema hematopoyético

STATI UNITI

Washington
ADDRESS: 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 IRDiRCHematopoietic stem cell biology
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

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

GIAPPONE

JAPAN
AKITA

Finanziato da un ente associato a IRDiRCIdentification of diagnostic biomarkers for refractory/relapsing acquired pure red cell aplasia (PRCA) by next generation sequencing
Akita University Graduate School of Medicine
Department of General Internal Medicine and Clinical Laboratory Medicine

Progetti di ricerca multicentrici