MCGOVERN MEDICAL SCHOOL’S BROWN FOUNDATION INSTITUTE of MOLECULAR MEDICINE FOR THE PREVENTION of HUMAN DISEASES IMMpact Report FISCAL YEAR 2017 About the cover It’s a beautiful day at McGovern Medical School’s Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases. IMMpact Report is published by McGovern Medical School. All correspondence should be addressed to: Office of Communications 6431 Fannin, B.340 Houston, TX 77030 E-mail: [email protected] Articles and photos may be reprinted with permission. Editor Darla Brown, Director, Office of Communications Contributors: Darla Brown Rob Cahill Jonathan Garris IMM Faculty Design: Roy Prichard Photography: Dwight Andrews Maricruz Kwon For information on supporting programs at the McGovern Medical School and the IMM, contact Lise Cameron, 713.500.3602, [email protected] OOC-ThomasPrintworks-1175-3/18 Contents 2 Director's Message Features 3 Mission 4 Our Locations 6 8 16 Center for Cardiovascular Genetics Gene editing – Moving promising it starts therapeutics into 20 Center for Human Genetics in the lab practice 25 Center for Immunology and Autoimmune Diseases 31 Center for Metabolic and Degenerative Diseases 40 Center for Molecular Imaging 11 12 Breathing new life Entering uncharted 48 Center for Precision Biomedicine into lung disease genome territory research in the fight 57 Center for Stem Cell and Regenerative Medicine against disease 74 Center for Tissue Engineering and Aging Research 81 Texas Therapeutics Institute 14 IMM 90 IMM Service Centers Cheering on the IMM PACT REPORT 92 By the Numbers 93 Gift Report • • • • 1 Director’s Message ’m pleased to introduce the latest annual IMMpact report for The Brown Foundation Institute of Molecular Medicine for the Prevention Iof Human Diseases (IMM). The IMM is a stand-alone research institute that is embedded within McGovern Medical School. The IMM mission is to deliver translational outcomes from research in molecular medicine that benefits patients. To this end, we have teams of outstanding basic and translational scientists who collaborate closely with our clinical colleagues. Inside the report you will find in-depth articles on some of our faculty and donors plus an account from each IMM faculty member describing their research programs. This year we have recruited additional outstanding new faculty, who bring with them exciting research ideas and innovative technologies. Two of our new recruits, whose stories are featured, also secured prestigious STARs awards from The University of Texas System, which are reserved for the recruitment of highly sought-after scientists. The environment for scientific research funding continues to be extremely challenging, especially The IMM has from the NIH. Despite this, IMM faculty have excelled again. Over the two major financial year just ended, our new grants and contracts were up some 30 percent over the preceding year, which in turn had seen a considerable objectives: increase over the prior year. It is a testament to the remarkable quality and Mission creativity of our scientists that the IMM remains so successful in attracting research funds from what is an ever-diminishing national pool. That Discovery is the 1 said, full implementation of our mission remains heavily dependent on he Brown Foundation research and the future practice to medical practice are of highest priority for attracting support from alternative sources, including research foundations, Institute of Molecular of medicine with more novel major importance to product the IMM faculty. This industry collaborations, and, most importantly, the continuing generosity Medicine for the therapies. These approaches development in biotechnology is a major challenge, of our friends and donors. T Prevention of Human Diseases since diabetes, obesity, In addition to advancing science and medicine, we wish to develop have been most successfully and the pharmaceutical cancer, Alzheimer’s, and our relationships with all in our community who value the aspiration (IMM) is a research institute used to determine the causes of industry, the IMM has cardiovascular diseases of our mission to translate molecular discoveries into new therapies for that seeks to investigate the infectious disorders and genetic the potential and desire to are unsolved medical human disease. In this regard, we are deeply appreciative of the strong causes of human diseases at diseases. form important links and work and dedication of the IMM advisory council, which plays a key role problems that are not the cellular and molecular However, it is clear that collaborations between its caused by single gene in the continued growth and development of the IMM. If you would levels, using DNA and protein molecular and cell biology own research activities and defects. Discoveries lead like to investigate how you can also be involved, I would be delighted to talk with you personally. Alternatively, I would be delighted to see you at technologies to elucidate disease will play a major role in various industries to apply its to new solutions. our annual IMMpact symposium. Last year 165 guests listened to three mechanisms. This development clarifying the causes of many discoveries and intellectual talks in the Beth Robertson Auditorium and attended a reception in New diagnostics and and progress are of particular unsolved problems of modern properties to pharmaceutical 2 the James T. Willerson, M.D. Discovery Hall. This year the symposium therapies are derivative will be held on April 18, 2018, and will feature talks on how to use new interest for future planning medicine, such as heart opportunities. of discovery and to the gene editing technologies to correct inherited diseases. The format will in the increasingly important disease, hypertension, vascular As an institute of McGovern benefit of patients. The be slightly different from previous years in that we will have an extended area of clinical research. The disorders, major mental Medical School, the Brown IMM focuses on these question time with an expert panel comprising the speakers plus UTHealth institute endeavors to design illnesses, and inflammatory Foundation Institute of medical solutions. The physicians. The symposium is an excellent opportunity to hear exciting methods of rational therapy and immunologic diseases. Molecular Medicine for the IMM has organized research stories directly from our faculty, to discuss its implications for talent in the Texas the future of medicine and health care, and to have all your questions and, wherever possible, The research of the institute’s Prevention of Human Diseases Therapeutics Institute answered. Full details are in this report; please mark the date in your strategies for the prevention of investigators is inspiring and strives to set the example IMM specifically to achieve calendar because it is a great opportunity to visit the IMM. human diseases. promises to fulfill the mission for research excellence and PACT REPORT this goal of patient Advances in molecular and of the IMM. collaboration locally, nationally, John Hancock, M.A., M.B., B.Chir., Ph.D., Sc.D. benefit from discovery. PACT REPORT Executive Director, Institute of Molecular Medicine cell biology have enormous Because the applications and internationally. IMM John S. Dunn Distinguished University Chair in Physiology and Medicine potential for innovative medical of molecular and cell biology • • • • • • • • 2 3 Our Locations e Brown Foundation Fayez S. Sarofim Research Building Institute of • Primary home of the IMM’s faculty, Molecular Medicine for the administration, and support staff. Prevention of Human Diseases • Located adjacent to the The University of Texas Health Science Center at Houston (UTHealth) University Center Tower within IMMpact the Texas Medical Center. • Opened in 2006, the building encompasses 255,748 gross square feet. Symposium South Campus Research Building – 3 (SCRB3) • SCRB3 is a collaboration between The University of Texas MD Anderson Cancer Wednesday Center and UTHealth, in cooperation with GE Healthcare and the Texas Enterprise Fund. April 18, 2018 • Six-stories, 315,000 square-feet located on the South Campus of the Texas Medical Center. 4:00-6:30 p.m. • Opened in 2009, this facility houses Positron Emission Tomography, Magnetic Resonance Imaging, Optical Imaging Tracers, a Fayez S. Sarofim Research Building Cyclotron, wet labs, and support offices. 1825 Pressler Street The Denton A. Cooley Building – Texas Heart Institute at St. Luke’s Episcopal Hospital Editing the Cut, Copy, Paste: Muscular • The IMM occupies a 31,000 square-foot high- Gene correction in Dystrophies: new tech laboratory. human Cystic Fibrosis hope from gene • Located in the Texas Medical Center. genome: the editing Brian Davis, PhD future is now Director of the Center for Stem Cell and Radbod Darabi, MD, PhD Regenerative Medicine Assistant Professor, Center for Stem Cell C. Harold and Lorine G. Wallace and Regenerative Medicine IMM Distinguished University Chair PACT REPORT PACT REPORT IMM • • • • • • • • 4 SAVE THE DATE 5 Profiles in research Gene editing – it starts in the lab ll green thumbs know that affects more than 30,000 specific cells (e.g. lung cells) that pruning is key to a people in the United States. The to that patient,” he explains. Ahealthy garden. Just as blood cells are from immune “This eliminates the need to use all good writers know that edit- deficiency patients who suffer donor stem cells from another ing keeps copy sharp. from recurrent infections. individual, minimizing the body Brian Davis, Ph.D., director “Our goal for the lung is to rejecting the cell.” of the IMM’s Center for Stem isolate, expand, and edit the In addition to creating stem Cell & Regenerative Medicine, stem cells as they exist in the cells, the tools used to specifi- uses these concepts of pruning airway and deliver them back cally correct gene defects are be- and editing in the laboratory healthy to the patient,” Dr. Da- coming more useful and precise. to modify genes – weeding out vis says. “The lung might not be “We can highlight an area to the errors in hopes of making the easiest place to reintroduce a cut and paste to make a specific patients healthy again.