Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system.

ABSTRACT: The preclinical development of microRNA-based gene therapies for inherited neurodegenerative diseases is accompanied by translational challenges. Due to the inaccessibility of the brain to periodically evaluate therapy effects, accessible and reliable biomarkers indicative of dosing, durability and therapeutic efficacy in the central nervous system are very much needed. This is particularly important for viral vector-based gene therapies, in which a one-time administration results in long-term expression of active therapeutic molecules in the brain. Recently, extracellular vesicles have been identified as carriers of RNA species, including microRNAs, and proteins in all biological fluids, whilst becoming potential sources of biomarkers for diagnosis. In this study, we investigated the secr

SUBMITTER: Sogorb-Gonzalez M 

PROVIDER: S-EPMC8093922 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature