Targeted gene transfer to the brain via the delivery of brain-penetrating DNA nanoparticles with focused ultrasound.

ABSTRACT: Gene therapy holds promise for the treatment of many pathologies of the central nervous system (CNS), including brain tumors and neurodegenerative diseases. However, the delivery of systemically administered gene carriers to the CNS is hindered by both the blood-brain barrier (BBB) and the nanoporous and electrostatically charged brain extracelluar matrix (ECM), which acts as a steric and adhesive barrier. We have previously shown that these physiological barriers may be overcome by, respectively, opening the BBB with MR image-guided focused ultrasound (FUS) and microbubbles and using highly compact "brain penetrating" nanoparticles (BPN) coated with a dense polyethylene glycol corona that prevents adhesion to ECM components. Here, we tested whether this combined approach could be utilized

SUBMITTER: Mead BP 

PROVIDER: S-EPMC4739627 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature