Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesions

CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy) is a hereditary small vessel disease caused by mutations in the NOTCH3 gene, characterized by recurrent strokes, cognitive decline, and psychiatric symptoms. This report presents a novel NOTCH3 c.156...

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Main Authors: Nicola Rifino, Silvia Baratta, Esteban Zacarias, Isabella Canavero, Benedetta Storti, Mario Stanziano, Emanuela Maderna, Gianluca Marucci, Franco Taroni, Anna Bersano
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Clinical Parkinsonism & Related Disorders
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Online Access:http://www.sciencedirect.com/science/article/pii/S2590112525000015
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author Nicola Rifino
Silvia Baratta
Esteban Zacarias
Isabella Canavero
Benedetta Storti
Mario Stanziano
Emanuela Maderna
Gianluca Marucci
Franco Taroni
Anna Bersano
author_facet Nicola Rifino
Silvia Baratta
Esteban Zacarias
Isabella Canavero
Benedetta Storti
Mario Stanziano
Emanuela Maderna
Gianluca Marucci
Franco Taroni
Anna Bersano
author_sort Nicola Rifino
collection DOAJ
description CADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy) is a hereditary small vessel disease caused by mutations in the NOTCH3 gene, characterized by recurrent strokes, cognitive decline, and psychiatric symptoms. This report presents a novel NOTCH3 c.1564 T > A (p.Cys522Ser) mutation associated with early-onset parkinsonism and significant white matter lesions. We describe a patient who presented with early-onset parkinsonism, characterized by bradykinesia and rigidity, alongside extensive white matter lesions observed through neuroimaging. Genetic testing revealed a novel c.1564 T > A (p.Cys522Ser) mutation in the NOTCH3 gene, contributing to the clinical diagnosis of CADASIL. This case underscores the phenotypic variability of CADASIL and the potential for atypical presentations, including parkinsonism. Early identification of genetic mutations can facilitate appropriate management and counseling for affected individuals and their families. Further research is warranted to explore the mechanisms underlying the association between NOTCH3 mutations and parkinsonism. Our findings contribute to the understanding of CADASIL, suggesting that clinicians should consider CADASIL in differential diagnoses of early-onset parkinsonism, especially in patients with concurrent white matter lesions.
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spelling doaj-art-e9d008c721d8493ea90cd96a48da56cd2025-08-20T02:34:46ZengElsevierClinical Parkinsonism & Related Disorders2590-11252025-01-011210029710.1016/j.prdoa.2025.100297Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesionsNicola Rifino0Silvia Baratta1Esteban Zacarias2Isabella Canavero3Benedetta Storti4Mario Stanziano5Emanuela Maderna6Gianluca Marucci7Franco Taroni8Anna Bersano9Cerebrovascular Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy; Corresponding author at: Via Celoria 11, 20134 Milan, Italy.Unit of Medical Genetics and Neurogenetics Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyCerebrovascular Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyCerebrovascular Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyCerebrovascular Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyNeuroradiology Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyNeuropathology Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyNeuropathology Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyUnit of Medical Genetics and Neurogenetics Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyCerebrovascular Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyCADASIL (Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy) is a hereditary small vessel disease caused by mutations in the NOTCH3 gene, characterized by recurrent strokes, cognitive decline, and psychiatric symptoms. This report presents a novel NOTCH3 c.1564 T > A (p.Cys522Ser) mutation associated with early-onset parkinsonism and significant white matter lesions. We describe a patient who presented with early-onset parkinsonism, characterized by bradykinesia and rigidity, alongside extensive white matter lesions observed through neuroimaging. Genetic testing revealed a novel c.1564 T > A (p.Cys522Ser) mutation in the NOTCH3 gene, contributing to the clinical diagnosis of CADASIL. This case underscores the phenotypic variability of CADASIL and the potential for atypical presentations, including parkinsonism. Early identification of genetic mutations can facilitate appropriate management and counseling for affected individuals and their families. Further research is warranted to explore the mechanisms underlying the association between NOTCH3 mutations and parkinsonism. Our findings contribute to the understanding of CADASIL, suggesting that clinicians should consider CADASIL in differential diagnoses of early-onset parkinsonism, especially in patients with concurrent white matter lesions.http://www.sciencedirect.com/science/article/pii/S2590112525000015CADASILParkinsonismStrokeVariant of uncertain significance
spellingShingle Nicola Rifino
Silvia Baratta
Esteban Zacarias
Isabella Canavero
Benedetta Storti
Mario Stanziano
Emanuela Maderna
Gianluca Marucci
Franco Taroni
Anna Bersano
Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesions
Clinical Parkinsonism & Related Disorders
CADASIL
Parkinsonism
Stroke
Variant of uncertain significance
title Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesions
title_full Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesions
title_fullStr Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesions
title_full_unstemmed Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesions
title_short Novel NOTCH3 mutation c.1564 T > A (p.Cys522Ser) presenting with early-onset Parkinsonism and white matter lesions
title_sort novel notch3 mutation c 1564 t a p cys522ser presenting with early onset parkinsonism and white matter lesions
topic CADASIL
Parkinsonism
Stroke
Variant of uncertain significance
url http://www.sciencedirect.com/science/article/pii/S2590112525000015
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