Gene therapy for cystic fibrosis lung disease: current status and future perspectives.

Cystic fibrosis (CF) is an autosomal recessive genetic disease affecting > 70,000 individuals worldwide. Despite improvements in current therapies, most patients do not survive beyond their early 30s. After cloning of the cystic fibrosis transmembrane regulator (CFTR) gene, there was considerable clinical interest in the possible therapeutic delivery of CFTR genes directly to the lung.

Interaction of polyamine gene vectors with RNA leads to the dissociation of plasmid DNA-carrier complexes.

Background: Plasmid DNA (pDNA) dissociation from polyamine gene vectors after cellular uptake has not been well characterized. A more detailed understanding of this process could lead to more efficient gene transfer agents. Since RNA is present in the cytoplasm at high concentrations and due to its structural similarity to DNA, we were interested in its conceivable interaction with polyamine gene vectors.

Insights into the mechanism of magnetofection using PEI-based magnetofectins for gene transfer.

Background: Gene delivery by the use of magnetic forces, so-called magnetofection, has been shown to enhance transfection efficiency of viral and non-viral systems up to several-hundred-fold. For this purpose gene carriers, such as polyethylenimine (PEI), are associated with superparamagnetic nanoparticles and complexed with plasmid DNA. Gene delivery is targeted by the application of a magnetic field.