VPS13C-associated Parkinson's disease: Two novel cases and review of the literature.


Journal

Parkinsonism & related disorders
ISSN: 1873-5126
Titre abrégé: Parkinsonism Relat Disord
Pays: England
ID NLM: 9513583

Informations de publication

Date de publication:
01 2022
Historique:
received: 28 09 2021
revised: 20 11 2021
accepted: 28 11 2021
pubmed: 8 12 2021
medline: 19 4 2022
entrez: 7 12 2021
Statut: ppublish

Résumé

VPS13C is a protein-coding gene involved in the regulation of mitochondrial function through the endolysosomal pathway in neurons. Homozygous and compound heterozygous VPS13C mutations are etiologically associated with early-onset Parkinson's disease (PD). Moreover, recent studies linked biallelic VPS13C mutations with the development of dementia with Lewy bodies (DLB). Neuropathological studies on two mutated subjects showed diffuse Lewy body disease. In this article, we report the clinical and genetic findings of two subjects affected by early-onset PD carrying three novel VPS13C mutations (i.e., one homozygous and one compound heterozygous), and review the previous literature on the genetic and clinical findings of VPS13C-mutated patients, contributing to the knowledge of this rare genetic alpha-synucleinopathy.

Identifiants

pubmed: 34875562
pii: S1353-8020(21)00438-7
doi: 10.1016/j.parkreldis.2021.11.031
pii:
doi:

Substances chimiques

Proteins 0
VPS13C protein, human 0

Types de publication

Letter Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

37-39

Informations de copyright

Copyright © 2021 Elsevier Ltd. All rights reserved.

Auteurs

Edoardo Monfrini (E)

Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Neurology Unit, Milan, Italy.

Francesca Spagnolo (F)

Neurological Department, Antonio Perrino's Hospital, Brindisi, Italy.

Margherita Canesi (M)

Department of Parkinson's Disease, Movement Disorders and Brain Injury Rehabilitation, 'Moriggia-Pelascini' Hospital, Gravedona ed Uniti, Como, Italy; Parkinson Institute, ASST G.Pini-CTO, Milan, Italy.

Agostino Seresini (A)

Medical Genetics Laboratory, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

Augusto Rini (A)

Neurological Department, Antonio Perrino's Hospital, Brindisi, Italy.

Bruno Passarella (B)

Neurological Department, Antonio Perrino's Hospital, Brindisi, Italy.

Marco Percetti (M)

Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy.

Manuela Seia (M)

Medical Genetics Laboratory, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

Stefano Goldwurm (S)

Parkinson Institute, ASST G.Pini-CTO, Milan, Italy.

Viviana Cereda (V)

Department of Parkinson's Disease, Movement Disorders and Brain Injury Rehabilitation, 'Moriggia-Pelascini' Hospital, Gravedona ed Uniti, Como, Italy; Parkinson Institute, ASST G.Pini-CTO, Milan, Italy.

Giacomo P Comi (GP)

Dino Ferrari Center, Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy; Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Neurology Unit, Milan, Italy.

Gianni Pezzoli (G)

Medical Genetics Laboratory, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

Alessio Di Fonzo (A)

Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Neurology Unit, Milan, Italy. Electronic address: alessio.difonzo@policlinico.mi.it.

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Classifications MeSH