PHARM 604Gene Therapy

Gene therapy offers extraordinary potential to treat both inherited and acquired diseases by using the body's own machinery to produce a therapeutic compound or protein, or up-regulate/down-regulate specific cellular processes. This course will examine the various extra- and intracellular barriers to DNA transfection, and the common vectors used in gene therapy. Various routes of administration, such as injected, inhaled, and transdermal will be discussed in terms of their challengers, and the successes of recent formulations described in the literature.