Lipid-based DNA therapeutics: hallmarks of non-viral gene delivery

J Buck, P Grossen, PR Cullis, J Huwyler… - ACS …, 2019 - ACS Publications
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 …

[HTML][HTML] Viral and nonviral delivery systems for gene delivery

N Nayerossadat, T Maedeh, PA Ali - Advanced biomedical …, 2012 - journals.lww.com
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 …

Lipid nanoparticle-delivered chemically modified mRNA restores chloride secretion in cystic fibrosis

E Robinson, KD MacDonald, K Slaughter, M McKinney… - Molecular Therapy, 2018 - cell.com
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 …

Nonviral vectors for gene delivery

MA Mintzer, EE Simanek - Chemical reviews, 2009 - ACS Publications
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 …

Microbial pathogenesis in cystic fibrosis: mucoid Pseudomonas aeruginosa and Burkholderia cepacia

JR Govan, V Deretic - Microbiological reviews, 1996 - Am Soc Microbiol
Respiratory infections with Pseudomonas aeruginosa and Burkholderia cepacia play a
major role in the pathogenesis of cystic fibrosis (CF). This review summarizes the latest …

Lipid nanoparticle technologies for nucleic acid delivery: A nanoarchitectonics perspective

AR Ferhan, S Park, H Park, H Tae… - Advanced Functional …, 2022 - Wiley Online Library
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 …

Pathophysiology and management of pulmonary infections in cystic fibrosis

RL Gibson, JL Burns, BW Ramsey - American journal of respiratory …, 2003 - atsjournals.org
This comprehensive State of the Art review summarizes the current published knowledge
base regarding the pathophysiology and microbiology of pulmonary disease in cystic …

Poly (ethylenimine) and its role in gene delivery

WT Godbey, KK Wu, AG Mikos - Journal of controlled release, 1999 - Elsevier
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 …

Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes

JO Rädler, I Koltover, T Salditt, CR Safinya - Science, 1997 - science.org
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 …

[HTML][HTML] Small interfering RNA for cancer treatment: overcoming hurdles in delivery

NB Charbe, ND Amnerkar, B Ramesh… - … Pharmaceutica Sinica B, 2020 - Elsevier
In many ways, cancer cells are different from healthy cells. A lot of tactical nano-based drug
delivery systems are based on the difference between cancer and healthy cells. Currently …