天美传媒

Donald B. Kohn, M.D., studies the biology of blood stem cells, which are located in the bone marrow and have two important properties: they can duplicate themselves and they can create all types of blood cells. Over the course of 30 years of research, Kohn has developed new clinical methods to treat genetic blood diseases using blood stem cells that have been modified to remove genetic mutations.

Kohn鈥檚 blood stem cell gene therapy method collects some of a patient鈥檚 own blood stem cells and either adds a good copy of the defective gene or fixes the broken genes to eliminate disease-causing mutations. The patient then receives a transplant of their own corrected stem cells, which will ideally create an ongoing supply of healthy blood cells. Importantly, this method eliminates the risk of rejection associated with receiving a bone marrow transplant from a different person, meaning the patient doesn鈥檛 have to take a lifelong supply of anti-rejection drugs. 

Kohn鈥檚 clinical trials for adenosine deaminase-deficient severe combined immunodeficiency (also known as ADA-SCID or bubble baby disease), a condition where babies are born without an immune system and often don鈥檛 survive past the first two years of life, have cured more than 40 babies to date. Babies with the condition and their families have traveled to UCLA for this life-saving treatment from as far away as Lebanon and a new company was formed in 2016 to further develop the therapy and make it available at other centers and to more patients. 

Kohn is now applying similar blood stem cell gene therapy techniques in clinical trials for two other diseases. One of these diseases is X-linked chronic granulomatous disease, a rare inherited immunodeficiency disorder that prevents white blood cells from effectively killing foreign invaders such as bacteria, fungi or other microorganisms. If untreated, patients often succumb to chronic granulomatous disease within the first decades of life. 

The second disease is sickle cell disease, the most common inherited blood disorder in the United States. This disease causes abnormal 鈥榮ickle-shaped鈥� red blood cells that block small blood vessels and do not provide the appropriate amount of oxygen to the body, resulting in debilitating pain and organ damage. Kohn鈥檚 clinical trial seeks to overcome or repair the genetic mutation that causes this devastating disease, which impacts millions worldwide. 

Kohn earned his bachelor鈥檚 and master鈥檚 degrees from the University of Illinois, Champaign-Urbana and his medical degree from the University of Wisconsin School Of Medicine. He completed a pediatric internship and residency in Wisconsin followed by a medical staff fellowship in the Lymphoid Malignancies Branch (formerly the Metabolism Branch) of the National Cancer Institute.

Kohn began working on gene therapy as a fellow at the National Institutes of Health in 1985 and then began practicing as a pediatric bone marrow transplant physician at Children鈥檚 Hospital Los Angeles in 1987.  While practicing at Children鈥檚 Hospital Los Angeles, he started his own lab focused on stem cell research and has continued this work, advancing new therapies from the lab to the clinic. 

Dr. Garc铆a-Sastre is a Professor in the Department of Microbiology and Director of the Global Health and Emerging Pathogens Institute of Icahn School of Medicine at Mount Sinai in New York. For the past 25 years, his research interest has been focused on the molecular biology of influenza viruses and several other negative-strand RNA viruses. During his post-doctoral training in the early 1990s, he developed, for the first time, novel strategies for expression of foreign antigens by a negative-strand RNA virus, influenza virus. He has made major contributions to the influenza virus field, including 1) the development of reverse genetics techniques allowing the generation of recombinant influenza viruses from plasmid DNA, (studies in collaboration with Dr. Palese); 2) the generation and evaluation of negative-strand RNA virus vectors as potential vaccine candidates against different infectious diseases, including malaria and AIDS, and 3) the identification of the biological role of the non-structural protein NS1 of influenza virus during infection: the inhibition of the type I interferon (IFN) system. His studies provided the first description and molecular analysis of a viral-encoded IFN antagonist among negative-strand RNA viruses.  These studies led to the generation of attenuated influenza viruses containing defined mutations in their IFN antagonist protein that might prove to be optimal live vaccines against influenza. His research has resulted in more than 480 scientific publications and reviews. Dr. Garc铆a-Sastre is the director of the Center for Research on Influenza Pathogenesis (CRIP), one of the five NIAID funded Centers of Excellence for Influenza Research and Surveillance. He was among the first members of the Vaccine Study Section and member of the Virology B Study Section of NIH. In addition, he has served for 5 years as Editor of Journal of Experimental Medicine, is Editor of PLoS Pathogens, Journal of Virology and Virus Research, and member of the Editorial Board of Virology, Vaccine, NPJ Vaccines and Influenza and Other Respiratory Diseases.  He is a member of the scientific advisory board of Keystone Symposia. He has been a co-organizer of the international course on Viral Vectors (2001), held in Heidelberg, Germany, sponsored by Federation of European Biochemical Societies (FEBS), and of the first Research Conference on Orthomyxoviruses in 2001, held in Texel, The Netherlands, sponsored by the European Scientific Working Group on Influenza (ESWI). He has also been a co-organizing of the 7th International Society for Vaccines meeting in 2013, and of Keystone Meetings in 2014 on Respiratory Virus Pathogenesis and in 2017 on Interferons. His publication in Science on the reconstruction and characterization of the pandemic influenza virus of 1918 has been awarded the distinction of the paper of the year 2005 by Lancet. In 2005, he became a Fellow of the American Academy of Microbiology, and in 2009, he received the Beijerink Professorship from the National Academy of Sciences of the Netherlands. In 2011, he has been elected President of the International Society for Vaccines, for 2014 and 2015. In 2017, he has been elected a fellow of the Royal Academy of Pharmacy in Spain.

Dr. Feilim Mac Gabhann is an associate professor of biomedical engineering at the Whiting School of Engineering and the Johns Hopkins School of Medicine. He is also a core faculty member of the Institute for Computational Medicine. 

His research focuses on microvascular development and remodeling. Dr. Mac Gabhann serves as the director of the Hopkins Office for Undergraduate Research.

His team is currently engaged in projects that include inhibiting vascular endothelial growth factor signaling in cancer and promoting vascular endothelial growth factor signaling in ischemic disease.

Dr. Mac Gabhann received his undergraduate degree in chemical engineering from University College Dublin. He earned his Ph.D. in biomedical engineering from Johns Hopkins. He completed his postdoctoral fellowship at the Cardiovascular Research Center at the University of Virginia before returning to Johns Hopkins to join the faculty.

His work has been recognized with numerous awards, including the NIH Pathway to Independence Award, the American Physiological Society Arthur C. Guyton Award for Excellence in Integrative Physiology and the Alfred P. Sloan Foundation Research Fellowship.

Dr. Jorge Nieva graduated from the University of California, Irvine College of Medicine in 1997, trained in internal medicine at University of California, San Diego and in oncology and hematology at the Scripps Clinic. In 2003 he joined the faculty of the Scripps Research Institute and the medical staff of the Scripps Clinic in La Jolla, CA. While at Scripps, Dr. Nieva pioneered new technology for the detection of cancer cells in the peripheral blood and discoveries related to the fundamental mechanisms of the immune system.
Dr. Nieva was recruited to the Billings Clinic in Montana in 2007 where he served as department chair and was a program leader who established the multidisciplinary lung cancer and head/neck cancer clinics at the cancer center. While in Billings, Dr. Nieva led efforts to establish a research program in virus-delivered cancer gene therapy and immunotherapy. His teams were awarded certificates for excellence in the conduct of cancer clinical trials from the National Cancer Institute and the American Society of Clinical Oncology. Dr. Nieva returned to California, joining the faculty of the Keck School of Medicine in 2014.