Neuroacanthocytosis Syndromes in an Italian Cohort: Clinical Spectrum, High Genetic Variability and Muscle Involvement.


Journal

Genes
ISSN: 2073-4425
Titre abrégé: Genes (Basel)
Pays: Switzerland
ID NLM: 101551097

Informations de publication

Date de publication:
26 02 2021
Historique:
received: 30 12 2020
revised: 17 02 2021
accepted: 22 02 2021
entrez: 3 3 2021
pubmed: 4 3 2021
medline: 4 8 2021
Statut: epublish

Résumé

Neuroacanthocytosis (NA) syndromes are a group of genetically defined diseases characterized by the association of red blood cell acanthocytosis, progressive degeneration of the basal ganglia and neuromuscular features with characteristic persistent hyperCKemia. The main NA syndromes include autosomal recessive chorea-acanthocytosis (ChAc) and X-linked McLeod syndrome (MLS). A series of Italian patients selected through a multicenter study for these specific neurological phenotypes underwent DNA sequencing of the

Identifiants

pubmed: 33652783
pii: genes12030344
doi: 10.3390/genes12030344
pmc: PMC7996727
pii:
doi:

Substances chimiques

VPS13A protein, human 0
Vesicular Transport Proteins 0

Types de publication

Clinical Trial Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

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Auteurs

Alessandro Vaisfeld (A)

Istituto di Medicina Genomica, Università Cattolica del Sacro Cuore, 00168 Roma, Italy.

Giorgia Bruno (G)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and Interuniversity Center for Research in Neurosciences, University of Campania "Luigi Vanvitelli", 80131 Naples, Italy.

Martina Petracca (M)

Fondazione Policlinico Universitario "A. Gemelli" IRCCS, UOC di Neurologia, 00168 Roma, Italy.

Anna Rita Bentivoglio (AR)

Fondazione Policlinico Universitario "A. Gemelli" IRCCS, UOC di Neurologia, 00168 Roma, Italy.
Dipartimento Universitario di Neuroscienze, Università Cattolica del Sacro Cuore, 00168 Rome, Italy.

Serenella Servidei (S)

Dipartimento Universitario di Neuroscienze, Università Cattolica del Sacro Cuore, 00168 Rome, Italy.
Fondazione Policlinico Universitario "A. Gemelli" IRCCS, UOC di Neurofisiopatologia, 00168 Rome, Italy.

Maria Gabriella Vita (MG)

Fondazione Policlinico Universitario "A. Gemelli" IRCCS, UOC di Neurologia, 00168 Roma, Italy.

Francesco Bove (F)

Fondazione Policlinico Universitario "A. Gemelli" IRCCS, UOC di Neurologia, 00168 Roma, Italy.
Dipartimento Universitario di Neuroscienze, Università Cattolica del Sacro Cuore, 00168 Rome, Italy.

Giulia Straccia (G)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and Interuniversity Center for Research in Neurosciences, University of Campania "Luigi Vanvitelli", 80131 Naples, Italy.

Clemente Dato (C)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and Interuniversity Center for Research in Neurosciences, University of Campania "Luigi Vanvitelli", 80131 Naples, Italy.

Giuseppe Di Iorio (G)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and Interuniversity Center for Research in Neurosciences, University of Campania "Luigi Vanvitelli", 80131 Naples, Italy.

Simone Sampaolo (S)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and Interuniversity Center for Research in Neurosciences, University of Campania "Luigi Vanvitelli", 80131 Naples, Italy.

Silvio Peluso (S)

Department of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, 80138 Naples, Italy.

Anna De Rosa (A)

Department of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, 80138 Naples, Italy.

Giuseppe De Michele (G)

Department of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, 80138 Naples, Italy.

Melissa Barghigiani (M)

Molecular Medicine, IRCCS Fondazione Stella Maris, 56128 Pisa, Italy.

Daniele Galatolo (D)

Molecular Medicine, IRCCS Fondazione Stella Maris, 56128 Pisa, Italy.

Alessandra Tessa (A)

Molecular Medicine, IRCCS Fondazione Stella Maris, 56128 Pisa, Italy.

Filippo Santorelli (F)

Molecular Medicine, IRCCS Fondazione Stella Maris, 56128 Pisa, Italy.

Pietro Chiurazzi (P)

Istituto di Medicina Genomica, Università Cattolica del Sacro Cuore, 00168 Roma, Italy.
Fondazione Policlinico Universitario "A. Gemelli" IRCCS, UOC Genetica Medica, 00168 Roma, Italy.

Mariarosa Anna Beatrice Melone (MAB)

Department of Advanced Medical and Surgical Sciences, 2nd Division of Neurology, Center for Rare Diseases and Interuniversity Center for Research in Neurosciences, University of Campania "Luigi Vanvitelli", 80131 Naples, Italy.
Sbarro Institute for Cancer Research and Molecular Medicine, Center for Biotechnology, Temple University, Philadelphia, PA 19122-6078, USA.

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Classifications MeSH