KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neurons

Mutations in the microtubule-binding motor protein kinesin 5 A (KIF5A) are implicated in several adult-onset motor neuron diseases, including Amyotrophic Lateral Sclerosis, Spastic Paraplegia Type 10 and Charcot-Marie-Tooth Disease Type 2. While KIF5 family members transport a variety of cargos alon...

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Main Authors: Irune Guerra San Juan, Jessie W. Brunner, Kevin Eggan, Ruud F. Toonen, Matthijs Verhage
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Neurobiology of Disease
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Online Access:http://www.sciencedirect.com/science/article/pii/S0969996124003619
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author Irune Guerra San Juan
Jessie W. Brunner
Kevin Eggan
Ruud F. Toonen
Matthijs Verhage
author_facet Irune Guerra San Juan
Jessie W. Brunner
Kevin Eggan
Ruud F. Toonen
Matthijs Verhage
author_sort Irune Guerra San Juan
collection DOAJ
description Mutations in the microtubule-binding motor protein kinesin 5 A (KIF5A) are implicated in several adult-onset motor neuron diseases, including Amyotrophic Lateral Sclerosis, Spastic Paraplegia Type 10 and Charcot-Marie-Tooth Disease Type 2. While KIF5 family members transport a variety of cargos along axons, the specific cargos affected by KIF5A mutations remain poorly understood. Here, we generated KIF5Anull mutant human motor neurons and analyzed the impact on axonal transport and motor neuron outgrowth and regeneration in vitro. KIF5A deficiency caused reduced neurite complexity in young neurons (DIV14) and defects in axonal regeneration. KIF5A deficiency did not affect neurofilament transport but impaired mitochondrial motility and anterograde speed at DIV42. Notably, KIF5A deficiency strongly reduced anterograde transport of splicing factor proline/glutamine-rich (SFPQ)-associated RNA granules in DIV42 axons. Hence, KIF5A plays a critical role in promoting axonal regrowth after injury and in driving the anterograde transport of mitochondria and especially SFPQ-associated RNA granules in mature neurons.
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spelling doaj-art-eca09670f2be4868b2736385fe27ea622025-08-20T02:26:27ZengElsevierNeurobiology of Disease1095-953X2025-01-0120410675910.1016/j.nbd.2024.106759KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neuronsIrune Guerra San Juan0Jessie W. Brunner1Kevin Eggan2Ruud F. Toonen3Matthijs Verhage4Department of Functional Genomics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam and VU Medical Center, Amsterdam, the Netherlands; Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA, USA; Department of Human Genetics, Amsterdam University Medical Center, Amsterdam, the Netherlands; Corresponding authors at: Department of Functional Genomics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam and VU Medical Center, Amsterdam, the Netherlands.Department of Functional Genomics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam and VU Medical Center, Amsterdam, the Netherlands; Department of Human Genetics, Amsterdam University Medical Center, Amsterdam, the NetherlandsDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA, USA; Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA, USADepartment of Functional Genomics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam and VU Medical Center, Amsterdam, the Netherlands; Department of Human Genetics, Amsterdam University Medical Center, Amsterdam, the NetherlandsDepartment of Functional Genomics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam and VU Medical Center, Amsterdam, the Netherlands; Department of Human Genetics, Amsterdam University Medical Center, Amsterdam, the Netherlands; Corresponding authors at: Department of Functional Genomics, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam and VU Medical Center, Amsterdam, the Netherlands.Mutations in the microtubule-binding motor protein kinesin 5 A (KIF5A) are implicated in several adult-onset motor neuron diseases, including Amyotrophic Lateral Sclerosis, Spastic Paraplegia Type 10 and Charcot-Marie-Tooth Disease Type 2. While KIF5 family members transport a variety of cargos along axons, the specific cargos affected by KIF5A mutations remain poorly understood. Here, we generated KIF5Anull mutant human motor neurons and analyzed the impact on axonal transport and motor neuron outgrowth and regeneration in vitro. KIF5A deficiency caused reduced neurite complexity in young neurons (DIV14) and defects in axonal regeneration. KIF5A deficiency did not affect neurofilament transport but impaired mitochondrial motility and anterograde speed at DIV42. Notably, KIF5A deficiency strongly reduced anterograde transport of splicing factor proline/glutamine-rich (SFPQ)-associated RNA granules in DIV42 axons. Hence, KIF5A plays a critical role in promoting axonal regrowth after injury and in driving the anterograde transport of mitochondria and especially SFPQ-associated RNA granules in mature neurons.http://www.sciencedirect.com/science/article/pii/S0969996124003619KIF5AKinesinsMotor neuronsHuman neuronsAxonal transportMitochondria
spellingShingle Irune Guerra San Juan
Jessie W. Brunner
Kevin Eggan
Ruud F. Toonen
Matthijs Verhage
KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neurons
Neurobiology of Disease
KIF5A
Kinesins
Motor neurons
Human neurons
Axonal transport
Mitochondria
title KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neurons
title_full KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neurons
title_fullStr KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neurons
title_full_unstemmed KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neurons
title_short KIF5A regulates axonal repair and time-dependent axonal transport of SFPQ granules and mitochondria in human motor neurons
title_sort kif5a regulates axonal repair and time dependent axonal transport of sfpq granules and mitochondria in human motor neurons
topic KIF5A
Kinesins
Motor neurons
Human neurons
Axonal transport
Mitochondria
url http://www.sciencedirect.com/science/article/pii/S0969996124003619
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