Kohn is a researcher with the UCLA Broad Stem Cell Research Center and a professor of microbiology, immunology and molecular genetics and pediatrics.
“The gene therapy field has matured considerably in the last decade,” said Kohn, who serves as director of the UCLA Human Gene Medicine Program, one of the largest in the nation. Using gene therapy, Kohn, a world leading expert in stem cell and translational medicine, believes he can target the root causes of these diseases, adding an improved gene that prevents red blood cells from “sickling,” putting back a missing gene that makes an enzyme to prevent severe combined immunodeficiency (SCID) or bubble boy syndrome, and engineering blood stem cells to make T-cells that can seek out and fight acute lymphocytic leukemia (ALL), chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphoma. Donald Kohn has a vision – if he can genetically fix the root causes of certain diseases from inside the dysfunctional cells, he may uncover new and more effective therapies for such devastating maladies as sickle cell disease, “bubble boy” syndrome, and virulent forms of cancer.Ĭurrent treatments for these disorders are ineffective, and do not seek to fix what’s wrong with the cells from the inside.
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