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Academic Background Dr. Emerson received his undergraduate education at Princeton University in Biology/Biochemistry, and his graduate training at MIT and the University of California, San Diego in Cell and Molecular Biology. He then pursued postdoctoral research as an American Cancer Society Postdoctoral Fellow at the University of Virginia, where he initiated his career-long studies of skeletal myogenesis. He received his first faculty appointment in the Department of Biology at the University of Virginia and advanced to become Commonwealth Professor of Biology. His subsequent faculty appointments include: Senior Scientist at Fox Chase Cancer Center, the Joseph Leidy Professor and Chair of Cell and Developmental Biology at the University of Pennsylvania School of Medicine, Director and Senior Scientist at the Boston Biomedical Research Institute, as well as visiting scientist at the Carnegie Institution Department of Embryology and the Pasteur Institute. Dr. Emerson joined the faculty of the University of Massachusetts Medical School in 2013 as Professor of Cell and Developmental Biology and Neurology and as Director of the Wellstone Muscular Dystrophy Program. At UMMS, he has continued his investigations of skeletal muscle development, focusing on human muscle biology and muscular dystrophies. His research has been generously supported by NIH, including Career Development and Merit Awards and directorship of an NIH Wellstone Muscular Dystrophy Cooperative Research Center, and by foundations supporting cancer and muscular dystrophy research. Throughout his career, he has had many valued and productive research collaborations, he has lead NIH graduate and postdoctoral training programs in cell and developmental biology, and he has been the proud mentor to a cadre of talented graduate students and postdoctoral fellows. Emerson Lab Research Program Skeletal muscle development, regeneration and disease Skeletal muscle is the most abundant tissue in the human body, responsible for all voluntary motor activity to enable our amazingly complex behaviors and adaptive functions- every smile, breath, and step. The Emerson Lab utilizes genome, molecular and stem cell technologies to understand how muscles develop in embryos and how injured muscles repair and regenerate in adults, with the goal of understanding molecular pathologies of human genetic muscular dystrophies and the development of therapeutics. Our lab is highly collaborative with academic and industry scientists, clinicians and patient advocates though the UMMS Wellstone Muscular Dystrophy Cooperative Research Center, with the support of NIH and foundations dedicated to the treatment of muscular dystrophies. Current Research iPSC modeling of human skeletal myogenesis and muscular dystrophy. Dr. Emerson’s research historically has utilized molecular, embryological and genetic approaches to investigate transcriptional and signaling regulation of skeletal muscle development in animal models including birds, Drosophia and mouse as well as cell culture myoblast models. Recently the Emerson lab has focused its research on human muscle biology, utilizing iPSC reprogramming and differentiation technologies to investigate the earliest epigenetic and molecular regulatory mechanisms that regulate commitment of pluripotent human cells to form muscle stem cell lineages, processes not readily accessible to investigation in embryos. These iPSC modelling studies have been made possible through collaboration with Genea Biocells, who are developing gene-free methods for step-wise chemical and growth factor induction of skeletal muscle stem cells from human embryonic stem cells (ESCs) and in the Emerson Lab, induced pluripotent stem cells (iPSCs), based on knowledge of signaling pathways operative during muscle development in the vertebrate embryo. These innovative ESC and iPSC skeletal muscle technologies have uniquely enabled the Emerson lab to investigate genetic and epigenetic regulatory mechanisms controlling the earliest stages of human skeletal muscle development as well as the molecular pathologies of human genetic muscular dystrophies, including facioscapulohumeral muscular dystrophy (FSHD) and limb girdle muscular dystrophies (LGMD) including LGMD2i and LGMD2g and develop small molecule and RNA therapeutics, and CRISPR gene correction and stem cell therapies to treat patients with these diseases. Ongoing research projects in the Emerson lab: • Identification of early developmental master regulatory genes controlling muscle stem cell lineage specification using CRISPR gene expression and editing technologies and small molecule developmental pathway activators and inhibitors, in collaboration with Scot Wolfe at UMMS and Genea Biocells; • Identification of iPSC muscle stem cell lineages to promote efficient skeletal muscle regeneration through development of muscle xenoengraftment technologies; • Modeling the role of innate immunity in FSHD muscle pathology, using combined muscle and blood xenografting technologies in immune deficient mice, in collaboration with Michael Brehm at UMMS; • Investigations of FSHD clinical disease severity through iPSC modeling of the molecular pathologies of iPSC muscle reprogrammed from FSHD patients with mild adult-onset disease and profound infantile-onset disease; • Development of RNA and small molecule therapeutics for FSHD, in collaboration with Jon Watts and Anastasia Khvorova at UMMS and Genea Biocells. • Development of CRISPR gene correction therapeutics for LGMD2i and LGMD2g in collaboration with Scot Wolfe at UMMS.
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