Assessment of CRISPRa-mediated gdnf overexpression in an In vitro Parkinson’s disease model

Abstract

The delivery system exhibits significant endosomal escape of more than 56%, crucial for the effective delivery and activation of the genetic material within cells. The increased <i>gdnf</i> expression correlates with a notable reduction in MAO-B complex activity, reaching basal values of 14.8 μU/μg of protein, and a reduction in reactive oxygen species. Additionally, there is up to a 34.6% increase in cell viability in an <i>In vitro</i> Parkinson's disease model treated with the neurotoxin MPTP. Our study shows that increasing <i>gdnf</i> expression can remediate some of the cellular symptoms associated with Parkinson's disease in an <i>in vitro</i> model of the disease using a novel nanostructured delivery system.