Noninvasive delivery of an α-synuclein gene silencing vector with magnetic resonance-guided focused ultrasound.

ABSTRACT:

Background

The characteristic progression of Lewy pathology in Parkinson's disease likely involves intercellular exchange and the accumulation of misfolded α-synuclein amplified by a prion-like self-templating mechanism. Silencing of the α-synuclein gene could provide long-lasting disease-modifying benefits by reducing the requisite substrate for the spreading aggregation.

Objectives

As a result of the poor penetration of viral vectors across the blood-brain barrier, gene therapy for central nervous system disorders requires direct injections into the affected brain regions, and invasiveness is further increased by the need for bilateral delivery to multiple brain regions. Here we test a noninvasive approach by combining low-intensity magnetic resonance-guided focused ultras

SUBMITTER: Xhima K 

PROVIDER: S-EPMC6282171 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature