Blood coagulation and thrombosis, gene-editing for treatment of diseases
My colleagues and I are interested in understanding the mechanisms of normal and abnormal blood coagulation, inflammation, and vascular biology. In particular, we focus on elucidating the pathogenesis of thrombotic thrombocytopenic purpura (TTP), a potentially fatal syndrome. Deficiency of plasma ADAMTS13 metalloprotease is the primary cause. ADAMTS13 is an enzyme that cleaves von Willebrand factor (VWF), an adhesion protein that helps agglutinate platelets for hemostasis. Deficiency of this enzyme leads to exaggerated platelet agglutination and the formation of microvascular thrombosis throughout the body, resulting in death. Current treatment is whole body plasma exchange. We are among the first group of investigators who first discovered and cloned ADAMTS13 protease. In the past decade, our lab has made major contribution in determining the structure-function relationship and cofactor-dependent regulation of ADAMTS13. Our lab is committed to develop novel tools for diagnosis and treatment of TTP. Potential rotation projects for graduate students include: a) structure-function relationship of ADAMTS13; b) cofactor-dependent regulation of ADAMTS13 function; c) novel assay development for diagnosis of TTP; d) the role of systemic inflammation in pathogenesis of TTP; e) AAV-mediated gene therapy for hereditary and acquired TTP; f) CRISPR/cas9 gene editing for correction of somatic mutations in diseases. Techniques in molecular biology, biochemistry/biophysics, and cell biology, as well as animal models are all commonly used in the laboratory. Current personnel: Wendy Cao, MD, PhD, Instructor; Vikram Pillai, PhD, Research V; Liang Zheng, PhD, Postdoctoral fellow; Dino Halkidis, MD, PhD, hem/onc fellow; Jean Sui, MD, hematologist visiting scholar, Di Zhang, MD, Pathologist visiting scholar; Mohammad Abdelgawwd, M.D., MS, graduate student, and Nicole Kocher, BS, research technician.
Contact Information
X. Long Zheng
xzheng@uabmc.edu
205-996-7451
