Introduction: The overexpression of transferrin (Tf) receptors on cancer cells renders them a useful target for the delivery of small-molecule drugs and nucleic acid therapeutics to these cells. This approach could alleviate the non-target effects of the drugs.
Areas covered: The function of the Tf receptor, the development of Tf-lipid-DNA complexes (Tf lipoplexes), therapeutic use of lipoplexes and polymer-DNA complexes (poylplexes), and the therapeutic use of Tf-lipoplexes and anti-Tf-receptor antibody-lipoplexes are outlined. The literature search for this review was based primarily on the terms 'lipoplexes,' 'lipopolyplexes' 'transferrin,' 'transferrin receptor,' and 'gene therapy.' However, the review was not intended to be comprehensive.
Expert opinion: Complexes of Tf with cationic liposomes and nucleic acids, or liposomes with covalently attached Tf or anti-transferrin receptor antibodies have been used for the delivery of therapeutic genes, antisense oligodeoxynucleotides, and short interfering RNA. Although such targeted nonviral delivery vehicles may benefit from further enhancement of their efficacy, current achievements at the cell culture and animal model level should be translated into clinical applications, restricted initially to localized delivery into accessible tissues to avoid potential systemic side-effects and non-target delivery.