Adding functional entities to plasmids.
Non-viral gene therapy constitutes an alternative to the more common use of viral-mediated gene transfer. Most gene transfer methods using naked DNA are based upon non-sequence-specific interactions between the nucleic acid and cationic lipids (lipoplex) or polymers (polyplex). We have developed a technology in which functional entities hybridize in a sequence-specific manner to the nucleic acid (bioplex). This technology is still in its infancy, but has the potential to become a useful tool, since it allows the construction of highly defined complexes containing a variety of functional entities. In its present form the bioplex technology is based upon the use of peptide/nucleic acids (PNA) as anchors. Single, or multiple, functional entities are directly coupled to the anchors. By designing plasmids, or oligonucleotides, with the corresponding anchor target sequence, complexes with desired composition can easily be generated. The long-term aim is to combine functional entities in order to achieve optimal, synergistic interactions allowing enhanced gene transfer in vivo.
Clinical Research Center, Karolinska Institutet, Huddinge University Hospital, SE-141 86 Stockholm, Sweden.
M G
MG
Svahn
K E
KE
Lundin
R
R
Ge
E
E
Törnquist
E O
EO
Simonson
S
S
Oscarsson
M
M
Leijon
L J
LJ
Brandén
C I E
CI
Smith
D002467:Cell Nucleus / Q000502:physiology; D018014:Gene Transfer Techniques; D005822:Genetic Vectors / Q000737:chemistry*; D020135:Peptide Nucleic Acids; D010957:Plasmids / Q000737:chemistry*; D021382:Protein Sorting Signals / Q000502:physiology
D020135:Peptide Nucleic Acids; D021382:Protein Sorting Signals