Therapeutic plasmid DNA versus siRNA delivery: common and different tasks for synthetic carriers

C Scholz, E Wagner - Journal of controlled release, 2012 - Elsevier
Gene therapy offers great opportunities for the treatment of severe diseases including
cancer. In recent years the design of synthetic carriers for nucleic acid delivery has become …

Cellular pathways of recombinant adeno-associated virus production for gene therapy

S Sha, AJ Maloney, G Katsikis, TNT Nguyen… - Biotechnology …, 2021 - Elsevier
Recombinant adeno-associated viruses (rAAVs) are among the most important vectors for in
vivo gene therapies. With the rapid development of gene therapy, current rAAV …

Mechanistic model for production of recombinant adeno-associated virus via triple transfection of HEK293 cells

TNT Nguyen, S Sha, MS Hong, AJ Maloney… - … Therapy Methods & …, 2021 - cell.com
Manufacturing of recombinant adeno-associated virus (rAAV) viral vectors remains
challenging, with low yields and low full: empty capsid ratios in the harvest. To elucidate the …

Synthetic incoherent feedforward circuits show adaptation to the amount of their genetic template

L Bleris, Z Xie, D Glass, A Adadey, E Sontag… - Molecular systems …, 2011 - embopress.org
Natural and synthetic biological networks must function reliably in the face of fluctuating
stoichiometry of their molecular components. These fluctuations are caused in part by …

[HTML][HTML] Single-cell mRNA transfection studies: delivery, kinetics and statistics by numbers

C Leonhardt, G Schwake, TR Stögbauer… - … , Biology and Medicine, 2014 - Elsevier
In artificial gene delivery, messenger RNA (mRNA) is an attractive alternative to plasmid
DNA (pDNA) since it does not require transfer into the cell nucleus. Here we show that …

Microfluidic formulation of lipid/polymer hybrid nanoparticles for plasmid DNA (pDNA) delivery

D Santhanes, A Wilkins, H Zhang, RJ Aitken… - International Journal of …, 2022 - Elsevier
Lipid/polymer hybrid nanoparticles loaded with red fluorescent protein (RFP) encoded
plasmid DNA (pDNA) was formulated using poly-lactic-co-glycolic acid (PLGA), cationic lipid …

Experiment and mathematical modeling of gene expression dynamics in a cell-free system

T Stögbauer, L Windhager, R Zimmer… - Integrative …, 2012 - academic.oup.com
Cell-free in vitro expression is increasingly important for high-throughput expression
screening, high yield protein production and synthetic biology applications. Yet its potential …

siRNA release kinetics from polymeric nanoparticles correlate with RNAi efficiency and inflammation therapy via oral delivery

C He, H Yue, L Xu, Y Liu, Y Song, C Tang, C Yin - Acta Biomaterialia, 2020 - Elsevier
Despite many efforts in the rational design of nanoparticles (NPs) to overcome the biological
barriers to small interfering RNA (siRNA) delivery for improving gene silencing efficiency …

Uptake and transfection efficiency of PEGylated cationic liposome–DNA complexes with and without RGD-tagging

RN Majzoub, CL Chan, KK Ewert, BFB Silva, KS Liang… - Biomaterials, 2014 - Elsevier
Steric stabilization of cationic liposome–DNA (CL–DNA) complexes is required for in vivo
applications such as gene therapy. PEGylation (PEG: poly (ethylene glycol)) of CL–DNA …

Rationally designed logic integration of regulatory signals in mammalian cells

M Leisner, L Bleris, J Lohmueller, Z Xie… - Nature …, 2010 - nature.com
Molecular-level information processing, is essential for 'smart'in vivo nanosystems. Natural
molecular computing, such as the regulation of messenger RNA (mRNA) synthesis by …