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. Mielcarek believes that individualizing treatment according to the patient's needs and philosophy, combined with incorporating up-to-date research knowledge, are key ingredients for excellent patient care.
Clinical Expertise

    Blood stem cell and bone marrow transplantation for hematologic malignancies
    Graft-versus-Host Disease (GVHD)

Title

    Medical Director, Adult Blood and Marrow Transplant Program, Seattle Cancer Care Alliance
    Member, Clinical Research Division, Fred Hutch
    Professor of Medicine, Department of Medical Oncology, University of Washington

Education and Training

    MD: Freie Universit盲t Berlin, 1986
    PhD: Freie Universit盲t Berlin, 1987
    Residency: Freie Universit盲t Berlin, Internal Medicine, 1987-1993
    Research Fellowship: Fred Hutchinson Cancer Research Center, 1994-1999
    Residency: University of Washington, Internal Medicine, 1999-2000
    Fellowship: University of Washington, Medical Oncology, 2000-2003

Owen graduated with an MRes in tissue engineering and regenerative medicine from the University of Manchester. He subsequently completed a PhD at the University of Birmingham that sought to compare the efficacy of stem cells isolated from adipose, bone marrow and dental pulp for the regeneration of mineralised tissues.

In 2016 Owen was awarded a competitive EPSRC E-TERM fellowship in collaboration with the University of Birmingham where he worked as an honorary visiting fellow in the School of Chemical Engineering with Professor Liam Grover. During his fellowship he pioneered the application of cell-derived nanoparticles, termed extracellular vesicles (EVs), for musculoskeletal therapies.

In April 2018 Owen was appointed as a lecturer in Molecular and Regenerative Biomedicine under the Loughborough Excellence 100 scheme. He was promoted to Senior Lecturer in 2021.

Miriam Merad, MD, PhD, is the Chair of the Department of Immunology and Immunotherapy, the Director of the Marc and Jennifer Lipschultz Precision Immunology Institute at Mount Sinai School of Medicine in New York and the Director of the Mount Sinai Human Immune Monitoring Center (HIMC).
Dr. Merad is an internationally acclaimed physician-scientist and a leader in the fields of dendritic cell and macrophage biology with a focus on their contribution to human diseases. Dr. Merad identified the tissue resident  macrophage lineage and revealed its distinct role in organ physiology and pathophysiology. She established the contribution of this macrophage lineage to cancer progression and inflammatory diseases and is now working on the development of novel macrophage-targeted therapies for these conditions. In addition to her work on macrophages, Dr. Merad is known for her work on dendritic cells, a group of cells that control adaptive immunity. She identified a new subset of dendritic cells, which is now considered a key target of antiviral and antitumor immunity.
Dr. Merad leads the Precision Immunology Institute at the Icahn School of Medicine (PrIISM) to bring immunology discoveries to the clinic. PrIISM integrates immunological research programs with synergistic expertise in biology, medicine, technology, physics, mathematics and computational biology to enhance our understanding of human immunology. She also founded the Human Immune Monitoring Center at Mount Sinai, one of the world’s most sophisticated research centers, which uses cutting-edge single-cell technology to understand the contribution of immune cells to major human diseases or treatment responses.
Dr. Merad has authored more than 200 primary papers and reviews in high profile journals. Her work has been cited several thousand times. She receives generous funding from the National Institutes of Health (NIH) for her research on innate immunity and their contribution to human disease, and belongs to several NIH consortia. She is an elected member of the American Society of Clinical Investigation and the recipient of the William B. Coley Award for Distinguished Research in Basic and Tumor Immunology. She is the President-elect of the International Union of Immunological Societies (IUIS). In 2020, she was elected to the National Academy of Sciences in recognition of her contributions to the field of immunology.
Please visit Dr. Merad's Lab website: 

Dr. Steve Stice is a University of Georgia, DW Brooks Distinguished Professor and Director of the Regenerative Bioscience Center, who holds a Georgia Research Alliance Eminent Scholar endowed chair, and is CSO of ArunA Biomedical Inc. He has over 30 years of research and development experience in biotechnology and is a co-founder of several biotech start-ups, including ArunA Biomedical; the first company to commercialize a product derived from human pluripotent stem cells, and cell development used to facilitate approval of Pfizer’s current cognitive enhancing pharmaceuticals.
Prior to joining UGA, Stice was the co-founder and served as both CSO and CEO of Advanced Cell Technology, the first USA Company to advance to human clinical trials using human pluripotent stem cells. Additionally, he co-founded startups; Prolinia and Cytogenesis which later merged with what is now, ViaCyte.
Outside of his academic professorship and business role, Stice co-directs The Regenerative Engineering and Medicine research center, or REM, a joint collaboration between Emory University, Georgia Institute of Technology and UGA, and is also a group leader of EBICS: Emergent Behaviors of Integrated Cellular Systems, a National Science Foundation Science and Technology Center founded by the Massachusetts Institute of Technology. As an invited member, he sits on the Scientific Advisory Board for the Food and Drug Administration (FDA), and is serving on the Governing Committee of the first institute funded by the U.S. Department of Commerce (DOC); National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), the eleventh institute in the Manufacturing USA government network.
Stice is a world-renown expert in the field of pluripotent stem cell biology. In 2001, he directed work on derivation of three human pluripotent stem cell lines which were approved for federal funding by the NIH and President Bush. One of several noteworthy achievements for Stice, was producing the first cloned rabbit in 1987 and the first cloned transgenic calves in 1998 (George and Charlie). In 1997 his group produced the first genetically modified embryonic stem cell derived pigs and cattle. Notably, the Stice lab was one of only five NIH sponsored sites for training NIH investigators on the propagation, differentiation and use of hESC over a six year period.
Currently, the Stice lab is developing novel therapies and new technologies for drug screening and neurodegenerative disease, which could change the lives of those suffering with Parkinson’s, Stroke injury, and Alzheimer’s. This research has led to publications in Science and Nature journals, national news coverage (CBS, NBC, ABC and CNN) and the first US patents on cloning animals and cattle stem cells which was featured in the Wall Street Journal. Most recently, Stice was elected to NAI Fellow status, the highest professional distinction accorded solely to academic inventors. Georgia Bio also honored Stice that same year with the 2017 Georgia Bio Industry Growth Award.

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