Design and development of polymers for gene delivery

DW Pack, AS Hoffman, S Pun, PS Stayton - Nature reviews Drug …, 2005 - nature.com
The lack of safe and efficient gene-delivery methods is a limiting obstacle to human gene
therapy. Synthetic gene-delivery agents, although safer than recombinant viruses, generally …

Synthetic DNA delivery systems

D Luo, WM Saltzman - Nature biotechnology, 2000 - nature.com
The ability to safely and efficiently transfer foreign DNA into cells is a fundamental goal in
biotechnology. Toward this end, rapid advances have recently been made in our …

The targeted delivery of multicomponent cargos to cancer cells by nanoporous particle-supported lipid bilayers

CE Ashley, EC Carnes, GK Phillips, D Padilla… - Nature materials, 2011 - nature.com
Encapsulation of drugs within nanocarriers that selectively target malignant cells promises to
mitigate side effects of conventional chemotherapy and to enable delivery of the unique drug …

mRNA as gene therapeutic: how to control protein expression

G Tavernier, O Andries, J Demeester… - Journal of controlled …, 2011 - Elsevier
For many years, it was generally accepted that mRNA is too unstable to be efficiently used
for gene therapy purposes. In the last decade, however, several research groups faced this …

Biodegradable nanoparticles for cytosolic delivery of therapeutics

JK Vasir, V Labhasetwar - Advanced drug delivery reviews, 2007 - Elsevier
Many therapeutics require efficient cytosolic delivery either because the receptors for those
drugs are located in the cytosol or their site of action is an intracellular organelle that …

Nonviral gene therapy: promises and challenges

S Li, L Huang - Gene therapy, 2000 - nature.com
The last 10 years have seen substantial progress in the development and application of
nonviral vectors in gene therapy. However, many problems remain to be resolved before …

Covalent linkage of the DNA repair template to the CRISPR-Cas9 nuclease enhances homology-directed repair

N Savic, FCAS Ringnalda, H Lindsay, C Berk… - elife, 2018 - elifesciences.org
The CRISPR-Cas9 targeted nuclease technology allows the insertion of genetic
modifications with single base-pair precision. The preference of mammalian cells to repair …

Insight into the mechanism of the peptide‐based gene delivery system MPG: implications for delivery of siRNA into mammalian cells

F Simeoni, MC Morris, F Heitz, G Divita - Nucleic acids research, 2003 - academic.oup.com
The improvement of non‐viral‐based gene delivery systems is of prime importance for the
future of gene and antisense therapies. We have previously described a peptide‐based …

Nanosystems in drug targeting: opportunities and challenges

JK Vasir, MK Reddy, VD Labhasetwar - Current Nanoscience, 2005 - ingentaconnect.com
The long cherished goal of targeting drugs to specific sites in the body, where the
pharmacological action is desired and sparing other tissues has been actively pursued all …

Formation and intracellular trafficking of lipoplexes and polyplexes

A Elouahabi, JM Ruysschaert - Molecular therapy, 2005 - cell.com
Cationic lipid/DNA lipoplexes and cationic polymer/DNA polyplexes represent an attractive
alternative to viral vectors for cell transfection in vitro and in vivo but still suffer from a …