Publication Date

12-30-2024

Journal

Nature Communications

DOI

10.1038/s41467-024-54982-9

PMID

39737939

PMCID

PMC11686342

PubMedCentral® Posted Date

12-30-2024

PubMedCentral® Full Text Version

Post-print

Published Open-Access

yes

Keywords

Mitochondrial Dynamics, Animals, Humans, Axotomy, Axons, Mice, Dynamins, Retinal Ganglion Cells, Induced Pluripotent Stem Cells, Apoptosis, Phosphorylation, MAP Kinase Kinase Kinases, Nerve Degeneration, Optic Nerve Injuries, Nerve Crush, Neurodegeneration, Cell death in the nervous system, Mechanisms of disease

Abstract

Currently there are no effective treatments for an array of neurodegenerative disorders to a large part because cell-based models fail to recapitulate disease. Here we develop a reproducible human iPSC-based model where laser axotomy causes retrograde axon degeneration leading to neuronal cell death. Time-lapse confocal imaging revealed that damage triggers an apoptotic wave of mitochondrial fission proceeding from the site of injury to the soma. We demonstrate that this apoptotic wave is locally initiated in the axon by dual leucine zipper kinase (DLK). We find that mitochondrial fission and resultant cell death are entirely dependent on phosphorylation of dynamin related protein 1 (DRP1) downstream of DLK, revealing a mechanism by which DLK can drive apoptosis. Importantly, we show that CRISPR mediated Drp1 depletion protects mouse retinal ganglion neurons from degeneration after optic nerve crush. Our results provide a platform for studying degeneration of human neurons, pinpoint key early events in damage related neural death and provide potential focus for therapeutic intervention.

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