Mostcurrent gene transfer methods function satisfactorily inspecialized systems involving established cell lines butareoften notapplicable withnonadherent, primary hematopoietic cells, which arenotoriously difficult totransfect. Toapproach this problem, wehaveinvestigated analternative method ofgenetransfer, "transferrnfection," inwhich DNA complexed totransferrin-poly cation conjugates isintroduced intocells byreceptor-mediated endocytosis (Wagner, E., Zenke, M.,Cotten, M.,Beug, H.& Birnstiel, M.L.(1990) Proc. Natl. Acad. Sci. USA87,3410-3414). Weshowherethat transferrin-polylysine andtransferrin-prot amine, whencom- plexed toplasmid DNAcontaining aluciferase reporter gene, isefficiently boundandmovedinto avian erythroblasts by endocytosis. Successful transfer andexpression oftheIu- ciferase reporter genedepends onspecific interaction ofthe transferrin-polylysine-DNA withthetransferrin re- ceptor andoccurs inasignificant fraction (>95%) ofthecells. Genetransfer efficiency bytransferrinfecti on islowerthan withanoptimized DEAE-dextran transfection methodbut reaches similar efficiencies whenthecells aretreated with chloroquine. Because theprocedure intheabsence ofchloro- quine iscompletely nontoxic tocells, aconstant expression level oftransferred genes maybemaintained byrepeated additions oftransferrin-poly lysine-DNA Inaddition, theuse-

Receptor-mediated endocytosis oftransferrin-poly cation conjugates: Anefficient waytointroduce DNA into hematopoietic cells

Cardiac xenotransplantation: recent preclinical progress with 3-month median survival.

Gene therapy is based on the assumption that a definitive cure for a genetic disease should be possible by directing treatment to the abnormal gene rather than to secondary effects of its products (Friedmann, 1989). Complementation of the altered gene can be obtained by introducing the normal gene into the cell genome by an appropriate vector. Retroviral vectors can be used to introduce exogenous DNA sequences into hematopoietic progenitors and pluripotent stem cells. Successful gene transfer has been reported in vitro and in vivo in the mouse, and with lower efficiency in hematopoietic progenitors derived from dogs and primates, including humans (Karlsson, 1991).

Progress Towards the Clinical Application of Somatic Cell Gene Therapy

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Progress Towards the Clinical Application of Somatic Cell Gene Therapy