AN EVALUATION OF RECEPTOR-MEDIATED GENE-TRANSFER USING SYNTHETIC DNA-LIGAND COMPLEXES

Receptor-mediated gene transfer is an attractive method for therapeuti cally correcting human genetic diseases since it permits the targeting of DNA to cellular receptors in specific tissues of adult animals. Ge nes introduced by this technique have been shown to be expressed in th e target tissue for varying periods. However, to be useful for gene th erapy, it is critical that both the chemical properties and physical i nteractions of the reagents involved in the design of the DNA delivery vehicle be rigorously characterized. In this review, we discuss the c ritical steps in the preparation of the DNA-ligand complex and the fac tors involved in the delivery and regulated expression of a transgene in animal tissues. The feasibility of using this technique for the the rapeutic delivery of genes to mammalian tissues will also be evaluated .