James F. Gusella, PhD

Bullard Professor of Neurogenetics
Department of Genetics
Harvard Medical School
Director, Center for Human Genetic Research
Massachusetts General Hospital
Associate Member
Broad Institute of MIT and Harvard
Principal Faculty
Harvard Stem Cell Institute

In 1980, Dr. James F. Gusella completed his PhD at M.I.T. where he showed that human DNA could be cloned based on its chromosomal location using recombinant libraries of somatic cell hybrids and hybridization with human-specific interspersed repeat DNA. Shortly before, Y.W. Kan had published his seminal paper describing a restriction fragment length polymorphism at the β-globin locus and concluded that “Polymorphism in a restriction
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Katherine A. High, MD

Co-founder, President and Chief Scientific Officer
Spark Therapeutics, Inc.

Dr. Katherine A. High is a hematologist with a longstanding interest in the development of gene therapy for the treatment of genetic disease. After a residency in internal medicine, she trained with Dr. Ed Benz as a fellow in hematology at Yale. She pursued a career in basic and translational research as a faculty member at UNC-Chapel Hill and at the University of Pennsylvania. Dr. High’s initial research interests focused on delineating the molecular defects responsible for hemophilia. Subsequently she sought to establish proof-of-concept in the canine model for a gene therapy approach to hemophilia, but was stymied by the inability to produce enough recombinant vector to treat a 20 kg dog. After a lunchtime
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Dennis Lo, MD, PhD

Chairman, Chemical Pathology
Director
Li Ka Shing Institute of Health Sciences
Associate Dean (Research), Faculty of Medicine
The Chinese University of Hong Kong

Dr. Dennis Lo is the Director of the Li Ka Shing Institute of Health Sciences of The Chinese University of Hong Kong. He received his undergraduate education from the University of Cambridge, and his Doctor of Medicine and Doctor of Philosophy degrees from the University of Oxford. Dennis Lo has been following Dr. Kan’s work since he was a medical student when he read an article titled ‘On a slow boat from China’, documenting Dr. Kan’s fascinating journey of science. Following Dr. Kan’s pioneering work in prenatal diagnosis, Dr. Lo worked in the area of non-invasive prenatal diagnosis. He discovered
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Robert L. Nussbaum, MD

Chief Medical Officer
Invitae
Clinical Professor
UCSF Department of Medicine
Member
UCSF Institute for Human Genetics

Dr. Robert L. Nussbaum is a leading human and clinical geneticist who specializes in the care of adults with hereditary disorders. His research has long focused on elucidating the genetic basis of disease, including Parkinson disease, Lowe syndrome, and many others. In 1993, Dr. Nussbaum helped launch the new intramural research program of what became the National Human Genome Research Institute. In 1996, he and his colleague Dr. Mihales Polymeropoulos mapped and identified the first hereditary form of Parkinson disease. Dr. Nussbaum was Chief of
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Stuart H. Orkin, MD

David G. Nathan Professor of Pediatrics
Harvard Medical School
Chairman, Pediatric Oncology
Dana-Farber Cancer Institute
Investigator, Pediatrics
Boston Children’s Hospital

Dr. Stuart H Orkin serves as Chairman of the Department of Pediatric Oncology at DFCI, the David G. Nathan Professor of Pediatrics at Harvard Medical School, and an Investigator of the Howard Hughes Medical Institute. In addition, he is a principal faculty member of the Harvard Stem Cell Institute (HSCI) and an associate of the Broad Institute of MIT/Harvard. He received his BS from MIT and MD from Harvard Medical School. He completed pediatric hematology/oncology training at Boston Children’s Hospital and the Dana Farber Cancer
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Griffin P. Rodgers, MD, MACP

Director
National Institute of Diabetes and Digestive and Kidney Diseases
Chief, Molecular and Clinical Hematology Branch
Section Chief, Molecular Hematology Section
National Institutes of Health

Dr. Griffin P. Rodgers, MD, MACP, was named director of the National Institute of Diabetes and Digestive and Kidney Diseases – part of the National Institutes of Health – in 2007. He provides scientific leadership and manages a staff of more than 600 employees and a budget of $2.0 billion. Dr. Rodgers is widely recognized for his contributions to the development of the first effective, and now FDA-approved, therapy for sickle cell anemia. Recently, he and his
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Bertram Lubin, MD

President & Chief Executive Officer
UCSF Benioff Children’s Hospital Oakland

In 1980, Dr. Lubin became the first director of medical research at Children’s Hospital Oakland, where he helped transform a small research program into Children’s Hospital Oakland Research Institute, a $50 million annual enterprise. In 2009 he was named President and CEO of the medical center, which recently affiliated with UCSF Benioff Children’s Hospital. Drs. Lubin and Kan first met in 1970 in Children’s Hospital Boston, where Kan was a junior faculty member and Lubin a hematology fellow. Both have spent their careers researching sickle cell anemia and advancing therapies.

Dr. Lubin’s complete biography is included in the symposium program.

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