We generated an iPSC line from a patient with spastic paraplegia type 10 (SPG10) carrying the novel missense variant c.50G > A (p.R17Q) in the N-terminal motor domain of the kinesin family member 5A (KIF5A) gene. This patient-derived in vitro cell model will help to investigate the role of different KIF5A mutations in inducing neurodegeneration in spastic paraplegia and in other KIF5A-related disorders, including Charcot-Marie-Tooth type 2 (CMT2) and amyotrophic lateral sclerosis (ALS).

Generation of an iPSC line from a patient with spastic paraplegia type 10 carrying a novel mutation in KIF5A gene / S. Santangelo, P. Bossolasco, S. Magri, C. Colombrita, S. Invernizzi, C. Gellera, L. Nanetti, D. Di Bella, V. Silani, F. Taroni, A. Ratti. - In: STEM CELL RESEARCH. - ISSN 1876-7753. - 66:(2023 Feb), pp. 103008.1-103008.5. [10.1016/j.scr.2022.103008]

Generation of an iPSC line from a patient with spastic paraplegia type 10 carrying a novel mutation in KIF5A gene

S. Santangelo
Primo
;
S. Invernizzi;V. Silani;A. Ratti
Ultimo
2023

Abstract

We generated an iPSC line from a patient with spastic paraplegia type 10 (SPG10) carrying the novel missense variant c.50G > A (p.R17Q) in the N-terminal motor domain of the kinesin family member 5A (KIF5A) gene. This patient-derived in vitro cell model will help to investigate the role of different KIF5A mutations in inducing neurodegeneration in spastic paraplegia and in other KIF5A-related disorders, including Charcot-Marie-Tooth type 2 (CMT2) and amyotrophic lateral sclerosis (ALS).
Settore MED/26 - Neurologia
Settore BIO/12 - Biochimica Clinica e Biologia Molecolare Clinica
feb-2023
21-dic-2022
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/955160
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