A Novel Protein Transduction Domain (Hph-1) as a Potential Nonviral Gene Delivery Carrier
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Gene therapy using nonviral gene delivery carriers has focused on the development and modification of synthetic carriers such as liposomes and polymers. Most polymers that are commercially used are taking advantage of their polycationic character which allows not only strong ligand-DNA affinity but also competent cell penetration. Despite the relatively high transfection efficiencies, high cytotoxicity is continuously pointed out as one of the major shortcomings of polycationic polymers such as polythyleneimine. Studies on the utilization of peptides have therefore been carried out recently to overcome these problems. For these reasons, the human transcription factor Hph-1, which is currently known as a protein transduction domain (PTD), has been studied for its potential to be used as a gene delivery carrier. Although its transfection efficiency has been about 10-fold lower than PEI, it has displayed almost no cytotoxicity even at concentrations as high as 100 M. As there are various methods reported that can enhance PTD’s transfection efficiency, the potential of Hph-1 to become an efficient gene delivery carrier superior to the commercial PEI is very high. This study serves as a preliminary evaluation of Hph-1 and encourages further investigation.
Abstract
Gene therapy using nonviral gene delivery carriers has focused on the development and modification of synthetic carriers such as liposomes and polymers. Most polymers that are commercially used are taking advantage of their polycationic character which allows not only strong ligand-DNA affinity but also competent cell penetration. Despite the relatively high transfection efficiencies, high cytotoxicity is continuously pointed out as one of the major shortcomings of polycationic polymers such as polythyleneimine. Studies on the utilization of peptides have therefore been carried out recently to overcome these problems. For these reasons, the human transcription factor Hph-1, which is currently known as a protein transduction domain (PTD), has been studied for its potential to be used as a gene delivery carrier. Although its transfection efficiency has been about 10-fold lower than PEI, it has displayed almost no cytotoxicity even at concentrations as high as 100 M. As there are various methods reported that can enhance PTD’s transfection efficiency, the potential of Hph-1 to become an efficient gene delivery carrier superior to the commercial PEI is very high. This study serves as a preliminary evaluation of Hph-1 and encourages further investigation.
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