(@freddigoldman)

Goal 2: Reduce Human Disease

How can we more safely deliver stem cells to Sickle Cell patients

Newer therapies using gene correction, rather than gene addition, are needed for sickle cell disease. Even with this potential advantage, there needs to be a way to safely deliver gene corrected HSC to the sickle cell patient. Chemotherapy is poorly tolerated, and often is the reason patients do not choose the BMT option. What is the status of other less toxic non myeloablative approaches, and how can they best be... more »

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51 net votes
67 up votes
16 down votes
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(@tosinola)

Goal 2: Reduce Human Disease

Impact of each VOC Crisis in patients with sickle cell disease

While the long term cumulative effects of frequency, duration and severity of VOC on mortality is known in SCD, there is little known about the impact of each individual crisis or the amount of damage during crisis versus background smoldering ischemia from the disease. Any effort in quantifying this for SCD in the absence of interventional agents initially, and then as a potential measurement of the benefit of drug... more »

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3 net votes
3 up votes
0 down votes
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(@rjjones)

Goal 3: Advance Translational Research

Allogeneic transplantation as a safe and universally available therapeutic strategy for treating non-malignant blood diseases

Can new advances in allogeneic blood or marrow transplantation (BMT) make the procedure a safe and universally available therapeutic strategy for treating non-malignant blood and immune disorders such as sickle cell anemia, thalassemia, aplastic anemia, and severe combined immune deficiency?

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164 net votes
214 up votes
50 down votes
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(@ewong0)

Goal 3: Advance Translational Research

Novel Cell Apheresis Technologies to Treat Hematologic Diseases

Current FDA approved apheresis technology uses elutriation/centrifugation or filtration separation techniques to remove pathologic cellular and/or plasma elements. Currently these techniques are non-specific, limited by inefficient removal kinetics and often require considerable blood product exposure. Despite tremendous improvement in our understanding of the pathophysiology of a variety of disease, our ability to... more »

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118 net votes
139 up votes
21 down votes
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(@jantin)

Goal 2: Reduce Human Disease

Transplantation across HLA barriers in aplastic anemia

Allogeneic stem cell transplantation is curative in aplastic anemia with much less intrinsic toxicity than transplantation in hematologic malignancies. The recent BMT-CTN trial demonstrated 97% survival at one year with little subsequent decline. However patients without matched related or unrelated donors have graft-rejection rates of up to 50%. Preliminary data from the Netherlands suggests that anti-thymocyte globulin... more »

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110 net votes
137 up votes
27 down votes
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(@smajumdar)

Goal 2: Reduce Human Disease

A Chidren's Oncology Group (COG) for sickle cell disease (SCD)?

We have all witnessed the success of the National Cancer Institute (NCI) funded Children's Oncology Group - an organization that has made tremendous advancements in the care of children with cancer, very rare compared to sickle cell disease. COG has been able to not only create a database of the numerous studies, but has the unique ability to make "smaller" institutions feel important as is evident by patient enrollment.... more »

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23 net votes
28 up votes
5 down votes
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