January 08, 2018
microRNAs (miRNAs) are critical for neuronal function and their dysregulation is repeatedly observed in neurodegenerative diseases. Here, we implemented high content image analysis for investigating the impact of several miRNAs in mouse primary motor neurons. This survey directed our attention to the neuron-specific miR-124, which controls axonal morphology. By performing next generation sequencing analysis and molecular studies, we characterized novel roles for miR-124 in control of mitochondria localization and function. We further demonstrated that the intermediate filament Vimentin is a key target of miR-124 in this system. Our data establishes a new pathway for control of mitochondria function in motor neurons, revealing the value of a neuron-specific miRNA gene as a mechanism for the re-shaping of otherwise ubiquitously-expressed intermediate filament network, upstream of mitochondria activity and cellular metabolism.
Publication - Summary
Apr 12, 2019
Journal of Controlled Release
Publication - Abstract
Aug 18, 2020
Nucleic Acids Research
In this paper Alnylam Pharmaceuticals, developer of RNAi therapeutics ONPATTRO® and GIVLAARI®, investigates the biological origins of the extended duration of N-acetylgalactosamine silencing RNA (GalNAc-siRNA) activity observed in vivo.