Highly PEGylated DNA Nanoparticles Provide Uniform and Widespread Gene Transfer in the Brain.

ABSTRACT: Gene delivery to the central nervous system (CNS) has potential as a means for treating numerous debilitating neurological diseases. Nonviral gene vector platforms are tailorable and can overcome key limitations intrinsic to virus-mediated delivery; however, lack of clinical efficacy with nonviral systems to date may be attributed to limited gene vector dispersion and transfection in vivo. It is shown that the brain extracellular matrix (ECM) strongly limits penetration of polymer-based gene vector nanoparticles (NP) through the brain parenchyma, even when they are very small (<60 nm) and coated with a polyethylene glycol (PEG) corona of typical density. Following convection enhanced delivery (CED), conventional gene vectors are confined to the injection site, presumably by adhesive intera

SUBMITTER: Mastorakos P 

PROVIDER: S-EPMC4433405 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature