Understanding how and why blood stem cells become cancerous is crucial in order to develop newer targeted therapies for acute myeloid leukemia (AML) and to prevent the onset of cancer following gene therapy. Research in my group will use knowledge gained from the study of healthy blood stem cells to better define the gene expression patterns that cause AML. This approach has already led to identification of an understudied gene that has a profound impact on the prognosis of AML patients and is required for the survival of AML cells in the lab. We will explore the role of this gene in regulating AML and develop tools to specifically block its function as a new class of targeted cancer drugs. ...

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Understanding how and why blood stem cells become cancerous is crucial in order to develop newer targeted therapies for acute myeloid leukemia (AML) and to prevent the onset of cancer following gene therapy. Research in my group will use knowledge gained from the study of healthy blood stem cells to better define the gene expression patterns that cause AML. This approach has already led to identification of an understudied gene that has a profound impact on the prognosis of AML patients and is required for the survival of AML cells in the lab. We will explore the role of this gene in regulating AML and develop tools to specifically block its function as a new class of targeted cancer drugs. We are also very interested in developing and optimizing gene therapies for blood-based disorders. Gene therapy offers a way to permanently cure an increasing number of genetic diseases, many of which can currently be treated only symptomatically. A major risk of some gene therapies is the development of secondary blood cancers months to years after gene therapy treatment, and we are developing tools to better understand and minimize that risk. As a model, we study a bone marrow failure disorder called Diamond-Blackfan anemia (DBA), which leads to insufficient numbers of red blood cells, and for which we have developed a gene therapy to increase red blood cell output. As development of cancer after gene therapy can be caused by the inappropriate activation of stem cell genes, we seek to optimize gene therapy enhancers that can restrict gene expression to only the red blood cells that are impacted in DBA. Support from CPRIT will enable me to launch my research program focusing on developing newer targeted ways to treat leukemia and better tools to prevent leukemia development after gene therapy. As current strategies to treat and prevent these cancers are insufficient, this work will have profound significance to patients across Texas, the United States, and the world.

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