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Harvard Department of
Stem Cell & Regenerative Biology (HSCRB)
Alexandra Glucksmann, Ph.D.
Chief Operating Officer
Senior R&D Scientist
Sigma-Aldrich Cell Design Studio
Associate Chief of Pathology & The Jim and Ann Orr Research Scholar,
Molecular Pathology Unit, Massachusetts General Hospital
Professor of Pathology, Harvard Medical School
Product Manager, Digital Biology Center
Bio-Rad Laboratories, Inc.
Feng Zhang Lab, Broad Institute/MIT
Harvard/MIT M.D.-Ph.D. program
MD in Health Sciences & Technology, MIT
Graduate Program in Biophysics, Harvard University
Chad Cowan received his BA and BS, with honors, from the University of Kansas. He received his PhD, from the University of Texas Southwestern at Dallas, garnering the Nominata award for most outstanding thesis. He subsequently completed a Damon Runyon postdoctoral fellowship with Professor Douglas Melton at Harvard University. He was named a Stowers Medical Investigator in 2006 and in 2008, he became an Assistant Professor at Harvard University. He is currently an Associate Professor at Harvard University in the Department of Stem Cell and Regenerative Biology and Massachusetts General Hospital, with appointments in the Center for Regenerative Medicine, Cardiovascular Research Center and Center for Human Genetics Research. He is an Associate Member of the Broad Institute and a Principal Faculty member of the Harvard Stem Cell Institute where he directs the Diabetes Disease Program and the iPS Cell Core Facility. Professor Cowan has led or been a member of several large efforts to utilize stem cells to better understand disease, including the NHLBI’s Next Gen iPS Cell Project and the Progenitor Cell Biology Consortium. In 2013, Professor Cowan received a Transformative Research Award from the NIH to create isogenic human pluripotent stem cell-based models of human disease mutations. More recently, Professor Cowan has focused on using genome-editing tools as therapeutics and as a co-founder of CRISPR Therapeutics hopes to see these discoveries translated into treatments or cures.
Modeling Cardiovascular and Metabolic Disease with Human Pluripotent Stem Cells
Our goal is to understand how naturally occurring human genetic variation protects (or predisposes) some people to cardiovascular and metabolic disease—the leading cause of death in the world—and to use that information to develop therapies that can protect the entire population from disease. Our strategy is to identify patients, families, and cohorts with disease; to use genetic techniques such as genome-wide association studies and exome sequencing to identify novel DNA variants and genes linked to disease; to use human cell-based models and mouse models to understand how the DNA variants affect gene and protein function; and to use these mechanistic insights to begin the process of developing new therapies that will benefit patients and populations. In particular, we are interested in using human pluripotent stem cells to create human-derived tissues, containing specific DNA variants, as genetic disease models in which environmental and epigenetic influences have been minimized. We also aim to use stem cells to enable regenerative medicine, in which a patient’s own cells can be genetically cured or made resistant to disease and then transplanted back into the body as a durable treatment.
Suzanne Hibbs received her BS from Missouri University of Science and Technology, and MSc from University of Missouri. She is currently a Senior R&D Scientist at Sigma-Aldrich where she utilizes cutting edge technology to develop custom cell lines in the Cell Design Studio team. She has nine years of industrial biotechnology experience, including product development and cell line engineering with ZFN, CRISPR and RNAi. Suzanne has broad cell biology background and extensive molecular genetics experience, including digital PCR assay development and screening for rare mutational events.
J. Keith Joung is Associate Chief of Pathology for Research and The Jim and Ann Orr Research Scholar at Massachusetts General Hospital (MGH) and a Professor of Pathology at Harvard Medical School. Dr. Joung has pioneered the development of important technologies for targeted genome and epigenome editing of human cells. He has received numerous awards including an NIH Director’s Pioneer Award, an NIH Director’s Transformative Research Project R01 Award, the Jim and Ann Orr MGH Research Scholar Award, and election into the American Association of University Pathologists. Dr. Joung holds a Ph.D. in genetics from Harvard University, an M.D. from Harvard Medical School and an A.B. in biochemical sciences from Harvard College.
Optimizing Engineered CRISPR-Cas9 Nucleases for Research and Therapeutic Applications
Yann Jouvenot, Ph.D. - Concurrently with developing new solutions for droplet digital PCR, Dr Jouvenot manages the development of new applications using the ddPCR technology. The analysis and quantification of genome editing events is part of the most recent applications. Before his current role, Dr Jouvenot was part of the R&D team exploring new applications. Prior to Bio-Rad, he gained extensive experience in the field of genome editing at Sangamo Biosciences, where he worked as a Scientist and helped to launch the CCR5 inactivation project for HIV cell therapy.
Winston Yan received his bachelor’s degree magna cum laude with high honors in physics from Harvard University in 2010. He is currently an MD/PhD student and Paul & Daisy Soros Fellow in the research group of Feng Zhang at the Broad Institute/MIT. Working to expand the CRISPR-Cas genome engineering toolbox, Winston hopes to develop more sophisticated in vivo models of human disease and pave the way towards therapeutic applications of genome engineering.
Efficient in vivo genome editing using Staphylococcus aureus Cas9
The RNA-guided endonuclease Cas9 has emerged as a versatile genome-editing platform. However, the size of the commonly used Cas9 from Streptococcus pyogenes (SpCas9) limits its ability to be packaged into the highly versatile adeno-associated virus (AAV) delivery vehicle. We characterize six smaller Cas9 orthologs and show that Cas9 from Staphylococcus aureus (SaCas9) can edit the genome with efficiencies similar to those of SpCas9. At >1kb shorter than SpCas9, SaCas9 can also be packaged with its sgRNA into a single AAV vector to allow efficient in vivodelivery. In targeting the liver, we demonstrate >40% editing of the cholesterol regulatory gene Pcsk9 in 1 week, resulting in a 40% decrease in total serum cholesterol. We furthermore assess the specificity of SaCas9 and SpCas9 using unbiased, genome-wide detection of double stranded breaks and show that SaCas9 can mediate genome editing in vivo with high specificity. The use of SaCas9 in AAV to efficiently and specifically edit the genome of cells in adult animals further expands the utility of CRISPR-Cas9 in basic research and therapeutic applications.
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Advancing Drug Discovery through Genome Engineering, is hosted by Merck Research Laboratories and the BioPharma Research Council. The BRC is an association for scientists across biomedical research - industry, academic, nonprofit, government, and supplier labs and their teams. To stimulate interactions that can lead to partnerships and collaborations, we provide conferences, symposia, roundtables, and webinars. Participants contribute by presenting their work, engaging in committees, and providing insights relevant to our dynamic community. www.biopharmaresearchcouncil.org