ABSTRACT

Two fields of intense research efforts, namely gene transfer and epidermal keratinocyte biology, have been united to provide a new avenue of somatic cell gene therapy. This chapter examines the choice of epidermal keratinocytes as recipient cells in gene therapy and their application in current research directed toward nonepidermal genetic diseases. It discusses additional approaches for gene therapy utilizing keratinocytes in treating genetically-based epidermal diseases. The chapter explores the feasibility of gene therapy using keratinocytes as a model system. The selection of epidermal keratinocytes as the cellular vehicle for gene therapy is supported when certain salient features of these cells and their tissue of origin are considered. A crucial prerequisite in determining whether keratinocytes might be suitable for gene therapy of systemic substances is verifying that keratinocytes can secrete factors that can transit the basement membrane and find their way to the bloodstream.