Gene therapy is a promising strategy for the treatment of monogenic disorders. Non-viral
gene delivery systems including lipid-based DNA therapeutics offer the opportunity to …

Gene therapy is the process of introducing foreign genomic materials into host cells to elicit
a therapeutic benefit. Although initially the main focus of gene therapy was on special …

The promise of gene therapy for the treatment of cystic fibrosis has yet to be fully clinically
realized despite years of effort toward correcting the underlying genetic defect in the cystic …

Gene therapy has gained significant attention over the past two decades as a potential
method for treating genetic disorders such as severe combined immunodeficiency, 1 cystic …

Respiratory infections with Pseudomonas aeruginosa and Burkholderia cepacia play a
major role in the pathogenesis of cystic fibrosis (CF). This review summarizes the latest …

This comprehensive State of the Art review summarizes the current published knowledge
base regarding the pathophysiology and microbiology of pulmonary disease in cystic …

Lipid‐based nanoparticles have emerged as a clinically viable platform technology to
deliver nucleic acids for a wide range of healthcare applications. Within this scope, one of …

Since the first published examination of poly (ethylenimine)(PEI) as a gene delivery vehicle,
there has been a flurry of research aimed at this polycation and its role in gene therapy …

Cationic liposomes complexed with DNA (CL-DNA) are promising synthetically based
nonviral carriers of DNA vectors for gene therapy. The solution structure of CL-DNA …

Cationic lipids are widely used for gene transfer in vitro and show promise as a vector for in
vivo gene therapy applications. However, there is limited understanding of the cellular and …