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Blood Diseases and Resources

Description:

The NHLBI Division of Blood Diseases and Resources (DBDR) plans and directs research and research training and career development programs, on the causes, prevention, and treatment of nonmalignant blood diseases, including anemias, sickle cell disease, and thalassemia; premalignant processes such as myelodysplasia and myeloproliferative disorders; hemophilia and other abnormalities of hemostasis and thrombosis; and immune dysfunction. Funding encompasses a broad spectrum of research ranging from basic biology to medical management of blood diseases. The Division has a major responsibility for research to improve the adequacy and safety of the nation's blood supply. It also plays a leading role in transfusion medicine research and applying stem cell biology to the development of new cell-based therapies to repair and regenerate human tissues and organs. The Division has three branches: the Blood Diseases Branch, the Thrombosis and Hemostasis Branch, and the Transfusion Medicine and Cellular Therapeutics Branch.

Blood Diseases Branch . Supports research and training for sickle cell disease, thalassemia, aplastic anemia and other red cell disorders from basic research on globin genes to clinical management.

Thrombosis and Hemostasis Branch. Supports research and training in occlusive disorders involved in deep vein thrombosis, in cardiovascular diseases and stroke, and in bleeding disorders.

Transfusion Medicine and Cellular Therapeutics Branch . Supports research and training in transfusion medicine, blood safety and resources, stem cell biology and disease, clinical cellular medicine; and Resource Programs that provide phenotypically-characterized biospecimens and GMP-grade cell therapies to the scientific community.

Research topics of interest to the Division of Blood Diseases and Resources include but are not limited to the following:

A. Animal models for blood diseases

1. Anemias including: sickle cell disease (development of larger animal models), thalassemia, Fanconi anemia, Diamond Blackfan anemia, and other anemias

2. Bleeding disorders including: hemophilia and von Willebrand disease

3. Inherited and acquired thrombocytopenias

4. Thrombosis and thrombolysis

5. Hereditary hemorrhagic telangiectasia

6. Paroxysmal nocturnal hemoglobinuria

7. Hemochromatosis

8. Myelodysplastic syndrome (MDS) and myeloproliferative disorders (MPD)

B. Animal models for complications associated with transfusion of blood products or cell-based therapies

1. Transfusion Related Acute Lung Injury (TRALI)

2. Transfusion-associated immuno and inflammatory complications including alloimmunization

3. Transfusion-transmitted infections such as Transmissible Spongiform Encephalopathy (TSE)

4. Graft versus Host Disease

5. Microoxygenation models to evaluate the effect of RBC transfusion

C. Animal models for the demonstration of safety and efficacy of novel cellular therapies including hemoglobin oxygen carriers (HBOC)

D. Tools, reagents, and assays for investigations of blood diseases and cellular therapies

1. Nanotechnologies

2. Proteomics

3. Glycomics

4. Genomics

5. Non-invasive approaches to analytes

E. Assays and technologies

1. Automated screening of therapeutic agents for blood diseases

2. Anti-thrombotic drug monitoring and thrombosis screening

3. Platelet functional tests

4. von Willebrand disease

5. Thrombotic Thrombocytopenia Purpura (TTP)

6. Multiplexed system for hemostatic factors, cytokines, and inflammatory agents

7. Non-invasive methodology to diagnose DVT and PE

8. Iron overload

9. Blood-borne infectious agents transmitted by blood transfusion, including agents causing babesiosis, dengue fever, malaria, and the transmissible spongiform encephalopathies such as variant Creutzfeldt-Jakob Disease (vCJD)

10. Diagnosis of inherited blood disorders

11. Information systems to manage and monitor continuous anti-coagulation

12. Prolonging the storage of transfusable blood components for therapeutic uses

13. Identification and characterization of microparticles and other bioactive substances in stored transfusable blood components

14. In vitro reduction, inactivation or removal of microorganisms and other infectious moieties from blood, blood components, and plasma derivatives

15. Platelet storage methods that preserve biological efficacy

16. Synthesizing, screening, and evaluating the safety and efficacy of therapeutic oxygen carriers

17. Synthesizing or purifying plasma proteins for therapeutic use

18. Measuring iron non-invasively

19. Non-invasive measurement of blood cell counts or other blood components

20. MHC haplotype determination by methods such as DNA fingerprinting techniques and single nucleotide polymorphisms

21. Tracking of engrafted cells using imaging and/or other techniques

22. Technologies to measure tissue microoxygenation

23. Development of HLA and HNA antibody assays

24. Cord blood collection devices

25. Microfluid assays for blood coagulation assessment

26. Quantitative technologies to predict engraftment of cell therapies including cord blood, peripheral blood and bone marrow

F. Technologies and strategies to improve blood donor screening practices

G. Drugs to Treat Hematologic Diseases and Cytopenic States

1. Anti-coagulants, including novel small molecule compounds

2. Specific agents to reverse the action of anti-coagulants

3. Oral anti-thrombotic agents

4. Dual action: anti-coagulants/anti-inflammatory agents

5. Anti-sickling agents or other pharmacologic approaches to the vasculopathy of sickle cell disease

6. Fetal hemoglobin enhancing agents

7. Fibrinolytic and anti-fibrinolytic agents

8. Iron chelation therapy including modification of existing agents to enhance efficacy

9. Replacement agents for hematologic factor deficiencies

H. Cellular Therapies

1. Expansion of cell populations including ex vivo expansion of cord blood, peripheral blood and bone marrow

2. Production and standardization of immune-modulating cytokines or monoclonal antibodies

3. Directed in vitro stem cell differentiation

4. Development of in vivo techniques to monitor survival, growth and development and differentiation of engrafted cells

5. Reprogramming differentiated cells to increase their lineage potential including the creation of induced pluripotent stem cells

I. Gene therapy vectors and delivery systems for the treatment of hematologic genetic diseases

J. Prothrombotic and hemorrhagic biomarkers and risk factors

K. Computational models for blood diseases and complications associated with transfusion of blood products and cellular therapies

L. Bioinformatics to store and analyze genes, proteins, and biomarkers for hemostasis

M. Equipment and procedures for the collection, separation, processing, preservation, storage, and distribution of blood and blood components and other cell therapies

N. Education

1. Patient and physician health education programs to improve patient management and to prevent or reduce the impact of blood diseases

2. Physician education programs to evaluate effectiveness and improve adherence to transfusion medicine clinical guidelines

3. Physician education materials to evaluate the effectiveness of cell therapies including cord blood, peripheral blood, and bone marrow transplantation

O. Public Health Education

1. Tutorials for community-based providers

2. Community health education programs in sickle cell disease, suitable for use in faith-based organizations or other community settings

3. Community health education programs to increase blood donation

P. Newborn Screening

1. Genetic counseling programs for families of infants with hemoglobinopathies or trait

2. Innovative data or systems to track follow-up and patient outcomes

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