Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegeneration

Abstract Mitochondrial proteins CHCHD10 and CHCHD2 are mutated in rare cases of heritable FTD, ALS and PD and aggregate in tissues affected by these diseases. Here, we show that both proteins form amyloid fibrils and report cryo-EM structures of fibrils formed from their disordered N-terminal domain...

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Main Authors: Guohua Lv, Nicole M. Sayles, Yun Huang, Chiara Mancinelli, Kevin McAvoy, Neil A. Shneider, Giovanni Manfredi, Hibiki Kawamata, David Eliezer
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-62149-3
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author Guohua Lv
Nicole M. Sayles
Yun Huang
Chiara Mancinelli
Kevin McAvoy
Neil A. Shneider
Giovanni Manfredi
Hibiki Kawamata
David Eliezer
author_facet Guohua Lv
Nicole M. Sayles
Yun Huang
Chiara Mancinelli
Kevin McAvoy
Neil A. Shneider
Giovanni Manfredi
Hibiki Kawamata
David Eliezer
author_sort Guohua Lv
collection DOAJ
description Abstract Mitochondrial proteins CHCHD10 and CHCHD2 are mutated in rare cases of heritable FTD, ALS and PD and aggregate in tissues affected by these diseases. Here, we show that both proteins form amyloid fibrils and report cryo-EM structures of fibrils formed from their disordered N-terminal domains. The ordered cores of these fibrils are comprised of a region highly conserved between the two proteins, and a subset of the CHCHD10 and CHCHD2 fibril structures share structural similarities and appear compatible with sequence variations in this region. In contrast, disease-associated mutations p.S59L in CHCHD10 and p.T61I in CHCHD2, situated within the ordered cores of these fibrils, cannot be accommodated by the wildtype structures and promote different protofilament folds and fibril structures. These results link CHCHD10 and CHCHD2 amyloid fibrils to neurodegeneration and further suggest that fibril formation by the WT proteins could also be involved in disease etiology.
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issn 2041-1723
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spelling doaj-art-3e555ce8420f4638b2b98ce106cbe72a2025-08-20T03:05:10ZengNature PortfolioNature Communications2041-17232025-08-0116111510.1038/s41467-025-62149-3Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegenerationGuohua Lv0Nicole M. Sayles1Yun Huang2Chiara Mancinelli3Kevin McAvoy4Neil A. Shneider5Giovanni Manfredi6Hibiki Kawamata7David Eliezer8Department of Biochemistry, Weill Cornell MedicineFeil Family Brain and Mind Research Institute, Weill Cornell MedicineDepartment of Physiology & Biophysics, Weill Cornell MedicineDepartment of Biochemistry, Weill Cornell MedicineFeil Family Brain and Mind Research Institute, Weill Cornell MedicineDepartment of Neurology, Center for Motor Neuron Biology and Disease, Columbia University Irving Medical CenterFeil Family Brain and Mind Research Institute, Weill Cornell MedicineFeil Family Brain and Mind Research Institute, Weill Cornell MedicineDepartment of Biochemistry, Weill Cornell MedicineAbstract Mitochondrial proteins CHCHD10 and CHCHD2 are mutated in rare cases of heritable FTD, ALS and PD and aggregate in tissues affected by these diseases. Here, we show that both proteins form amyloid fibrils and report cryo-EM structures of fibrils formed from their disordered N-terminal domains. The ordered cores of these fibrils are comprised of a region highly conserved between the two proteins, and a subset of the CHCHD10 and CHCHD2 fibril structures share structural similarities and appear compatible with sequence variations in this region. In contrast, disease-associated mutations p.S59L in CHCHD10 and p.T61I in CHCHD2, situated within the ordered cores of these fibrils, cannot be accommodated by the wildtype structures and promote different protofilament folds and fibril structures. These results link CHCHD10 and CHCHD2 amyloid fibrils to neurodegeneration and further suggest that fibril formation by the WT proteins could also be involved in disease etiology.https://doi.org/10.1038/s41467-025-62149-3
spellingShingle Guohua Lv
Nicole M. Sayles
Yun Huang
Chiara Mancinelli
Kevin McAvoy
Neil A. Shneider
Giovanni Manfredi
Hibiki Kawamata
David Eliezer
Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegeneration
Nature Communications
title Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegeneration
title_full Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegeneration
title_fullStr Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegeneration
title_full_unstemmed Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegeneration
title_short Amyloid fibril structures link CHCHD10 and CHCHD2 to neurodegeneration
title_sort amyloid fibril structures link chchd10 and chchd2 to neurodegeneration
url https://doi.org/10.1038/s41467-025-62149-3
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