Novel SPTBN2 gene mutation and first intragenic deletion in early onset spinocerebellar ataxia type 5.


Journal

Annals of clinical and translational neurology
ISSN: 2328-9503
Titre abrégé: Ann Clin Transl Neurol
Pays: United States
ID NLM: 101623278

Informations de publication

Date de publication:
04 2021
Historique:
revised: 03 03 2021
received: 11 02 2021
accepted: 03 03 2021
pubmed: 24 3 2021
medline: 12 1 2022
entrez: 23 3 2021
Statut: ppublish

Résumé

In the present study, we describe two novel cases of SCA5 with early onset. The first one, carrying a novel heterozygous de novo missense mutation in SPTBN2 gene, showed a striking very severe cerebellar atrophy and reduction of volume of the pons at a very young age (16 months). The latter, carrying the first de novo intragenic deletion so far reported in SPTBN2 gene, showed a mild cerebellar atrophy involving the hemispheres and a later onset. In both cases, for the first time, a hyperintense signal of the dentate nuclei was observed.

Identifiants

pubmed: 33756041
doi: 10.1002/acn3.51345
pmc: PMC8045899
doi:

Substances chimiques

SPTBN2 protein, human 0
Spectrin 12634-43-4

Types de publication

Case Reports Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

956-963

Informations de copyright

© 2021 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.

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Auteurs

Romina Romaniello (R)

Neuropsychiatry and Neurorehabilitation Unit, Scientific Institute, IRCCS Eugenio Medea, Bosisio Parini, Italy.

Andrea Citterio (A)

Laboratory of Molecular Biology, Scientific Institute, IRCCS Eugenio Medea, Bosisio Parini, Italy.

Elena Panzeri (E)

Laboratory of Molecular Biology, Scientific Institute, IRCCS Eugenio Medea, Bosisio Parini, Italy.

Filippo Arrigoni (F)

Neuroimaging Lab, Scientific Institute, IRCCS Eugenio Medea, Bosisio Parini, Lecco, Italy.

Marta De Rinaldis (M)

Unit for Severe Disabilities in Developmental Age and Young Adults, Scientific Institute, IRCCS E. Medea, Brindisi, Italy.

Antonio Trabacca (A)

Unit for Severe Disabilities in Developmental Age and Young Adults, Scientific Institute, IRCCS E. Medea, Brindisi, Italy.

Maria Teresa Bassi (MT)

Laboratory of Molecular Biology, Scientific Institute, IRCCS Eugenio Medea, Bosisio Parini, Italy.

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Classifications MeSH