Document Type

Article

Publication Date

2023

Keywords

JMG, SS1

JAX Source

Proc Natl Acad Sci U S A. 2023;120(44):e2313010120.

PMID

37878717

DOI

https://doi.org/10.1073/pnas.2313010120

Grant

We also thank the Genome Engineering Technologies service at The Jackson Laboratory (supported by NCI P30 CA034196). This work was supported by NIH grants to Y.C.W. (NINDS R00 NS109252), T.B.B. (NINDS Diversity Research Supplement 3R00NS109252- 04S2), R.W.B. (NINDS R24 NS098523 and R37 NS054154), and D.M.M. (R01 NS104295-01, NIH HEAL initiative supplement R01 NS104295-01 and R01 AR077691-01).

Abstract

Inter-organelle contact sites between mitochondria and lysosomes mediate the crosstalk and bidirectional regulation of their dynamics in health and disease. However, mitochondria-lysosome contact sites and their misregulation have not been investigated in peripheral sensory neurons. Charcot-Marie-Tooth type 2B disease is an autosomal dominant axonal neuropathy affecting peripheral sensory neurons caused by mutations in the GTPase Rab7. Using live super-resolution and confocal time-lapse microscopy, we showed that mitochondria-lysosome contact sites dynamically form in the soma and axons of peripheral sensory neurons. Interestingly, Charcot-Marie-Tooth type 2B mutant Rab7 led to prolonged mitochondria-lysosome contact site tethering preferentially in the axons of peripheral sensory neurons, due to impaired Rab7 GTP hydrolysis-mediated contact site untethering. We further generated a Charcot-Marie-Tooth type 2B mutant Rab7 knock-in mouse model which exhibited prolonged axonal mitochondria-lysosome contact site tethering and defective downstream axonal mitochondrial dynamics due to impaired Rab7 GTP hydrolysis as well as fragmented mitochondria in the axon of the sciatic nerve. Importantly, mutant Rab7 mice further demonstrated preferential sensory behavioral abnormalities and neuropathy, highlighting an important role for mutant Rab7 in driving degeneration of peripheral sensory neurons. Together, this study identifies an important role for mitochondria-lysosome contact sites in the pathogenesis of peripheral neuropathy.

Comments

Copyright © 2023 the Author(s). Published by PNAS. This open access article is distributed under Creative Commons Attribution-NonCommercial-NoDerivatives License 4.0 (CC BY-NC-ND).

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