Systemic Exosomal Delivery of shRNA Minicircles Prevents Parkinsonian Pathology.

ABSTRACT: The development of new therapies to slow down or halt the progression of Parkinson's disease is a health care priority. A key pathological feature is the presence of alpha-synuclein aggregates, and there is increasing evidence that alpha-synuclein propagation plays a central role in disease progression. Consequently, the downregulation of alpha-synuclein is a potential therapeutic target. As a chronic disease, the ideal treatment will be minimally invasive and effective in the long-term. Knockdown of gene expression has clear potential, and siRNAs specific to alpha-synuclein have been designed; however, the efficacy of siRNA treatment is limited by its short-term efficacy. To combat this, we designed shRNA minicircles (shRNA-MCs), with the potential for prolonged effectiveness, and used RV

SUBMITTER: Izco M 

PROVIDER: S-EPMC6904801 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature