Goal 3: Advance Translational Research

Genome Editing and Gene Therapy

There is a critical need for the establishment of strategies that will determine the efficacy, safety, and toxicity of genome editing techniques specifically in hematologic diseases.

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(@barbarak)

Goal 3: Advance Translational Research

Immune-Mediated hematologic disorders

What is the optimal approach to prevent and treat immune mediated hematologic disorders (autoimmune hemolytic anemia, immune thrombocytopenic purpura, etc) and complications of hematologic disease (inhibitors in hemophilia, transfusion-related alloimmunization, etc)

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(@ragni01)

Goal 3: Advance Translational Research

Novel Mechanism for Clinical Trials of New Pro-Hemostatic Agents in Hemophilia

There are new exciting novel pro-hemostatic therapeutics in early phase clinical trials for hemophilia and hemophilia inhibitor patients. Yet, it is difficult to design randomized trials to compare these agents, or compare them with standard treatment, given the small sample size and competing studies for such patients. It is critical to develop novel approaches to compare new agents in rare populations. For example,... more »

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6 net votes
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