Nonviral gene delivery: what we know and what is next

X Gao, KS Kim, D Liu - The AAPS journal, 2007 - Springer
Gene delivery using nonviral approaches has been extensively studied as a basic tool for
intracellular gene transfer and gene therapy. In the past, the primary focus has been on …

Hydrodynamic gene delivery: its principles and applications

T Suda, D Liu - Molecular Therapy, 2007 - cell.com
Efficient and safe methods for delivering genetic materials into cells must be developed
before the clinical potential of gene therapy can be fully realized. Recently, hydrodynamic …

Gene therapy progress and prospects: electroporation and other physical methods

DJ Wells - Gene therapy, 2004 - nature.com
Over the last 5 years, physical methods of plasmid delivery have revolutionized the
efficiency of nonviral gene transfer, in some cases reaching the efficiencies of viral vectors …

Alpha-1 antitrypsin deficiency: outstanding questions and future directions

M Torres-Durán, JL Lopez-Campos… - Orphanet journal of rare …, 2018 - Springer
Background Alpha-1 antitrypsin deficiency (AATD) is a rare hereditary condition that leads to
decreased circulating alpha-1 antitrypsin (AAT) levels, significantly increasing the risk of …

Hydroporation as the mechanism of hydrodynamic delivery

G Zhang, X Gao, YK Song, R Vollmer, DB Stolz… - Gene therapy, 2004 - nature.com
We have reported that a rapid tail vein injection of a large volume of plasmid DNA solution
into a mouse results in high level of transgene expression in the liver. Gene transfer …

Delivery of mRNA therapeutics for the treatment of hepatic diseases

Z Trepotec, E Lichtenegger, C Plank, MK Aneja… - Molecular Therapy, 2019 - cell.com
Promising improvements in the field of transcript therapeutics have clearly enhanced the
potential of mRNA as a new pillar for protein replacement therapies. Synthetic mRNAs are …

Gene therapy progress and prospects: hydrodynamic gene delivery

H Herweijer, JA Wolff - Gene therapy, 2007 - nature.com
Over the last few years, hydrodynamic tail vein delivery has established itself as a simple,
yet very effective method for gene transfer into small rodents. Hydrodynamic delivery of …

Hybrid superparamagnetic iron oxide nanoparticle-branched polyethylenimine magnetoplexes for gene transfection of vascular endothelial cells

R Namgung, K Singha, MK Yu, S Jon, YS Kim, Y Ahn… - Biomaterials, 2010 - Elsevier
The work demonstrated the development of thermally cross-linked superparamagnetic
nanomaterial which possessed polyethylene glycol moiety and covalently linked branched …

Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation

ZY Chen, E Riu, CY He, H Xu, MA Kay - Molecular Therapy, 2008 - cell.com
Minicircle DNA vectors devoid of plasmid bacterial backbone,(BB) DNAs, are
transcriptionally persistent, whereas their parent plasmid counterparts are silenced in the …

Structural impact of hydrodynamic injection on mouse liver

T Suda, X Gao, DB Stolz, D Liu - Gene therapy, 2007 - nature.com
The impact of hydrodynamic injection on liver structure was evaluated in mice using various
microscopic techniques. Upon hydrodynamic injection of approximately 9% of body weight …