Format

Send to

Choose Destination
Nanomed Nanosci Res. 2018;2(6). pii: 155. doi: 10.29011/2577-1477.100055. Epub 2018 Nov 23.

Optimizing Nanoparticle Design for Gene Therapy: Protection of Oligonucleotides from Degradation Without Impeding Release of Cargo.

Author information

1
Department of Neurology College of Medicine, University of South Florida, Tampa, FL, USA.

Abstract

Gene therapy delivery systems that rely on synthetic nanocarriers can be optimized by assays of nucleic acid protection and kinetic studies of nucleic acid release. These empirical measurements ensure nanoparticle stability and predict potential in vivo efficacy. Quantitative methods for assessment of the capacity of nanoparticles to protect oligonucleotide cargo and to measure the rate of release of the cargo were developed and tested based on six commercial cationic matrices. in vitro study of drug release kinetics provides predictable release rates under a variety of conditions which can be adapted to appropriate physiological factors that affect release in vivo. In brief, in vitro DNA release and DNase I degradation assays described here will be useful for optimization of nanocarrier-mediated gene therapy administration by various routes.

KEYWORDS:

Gene Therapy; Nanoparticles; Protection of Nucleic Acid Cargo; Release of Nucleic Acids from Nanoparticles

Supplemental Content

Full text links

Icon for PubMed Central
Loading ...
Support Center