Dr. Dieter Gruenert, Professor at the University of California, San Francisco, Department of Stomatology, spoke about the current goals and unknowns of gene therapy research. Dr. Gruenert has conducted gene therapy studies, some of them funded by CFRI. He described four goals of gene therapy research:
These goals cannot be achieved without considering which type of therapeutic DNA might be employed: integrated (disrupts other cells) or episomal (these genes float in a nucleus but do not disrupt other genes in the cells). Additionally, the type of protocol or delivery vehicle used to transport the genes to the CF cells is still uncertain. Both adenoviruses (modifying a common-cold virus to act as the delivery mechanism) or liposome vehicles are being considered. Finally there are safety issues which include the host immune response, cell inappropriate expression (the expression might occur in cells that normally do not express themselves) and pharmacotoxicity.
Seven active clinical trials are being conducted: five in the U.S. and two in Europe. During the question and answer period, Dr. Gruenert indicated that the next target for study in gene trials once the lungs have been thoroughly studied would be the liver.
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