Whole-Exome Sequencing and C9orf72 Analysis in Primary Progressive Aphasia.


Journal

Journal of Alzheimer's disease : JAD
ISSN: 1875-8908
Titre abrégé: J Alzheimers Dis
Pays: Netherlands
ID NLM: 9814863

Informations de publication

Date de publication:
2021
Historique:
pubmed: 23 2 2021
medline: 15 9 2021
entrez: 22 2 2021
Statut: ppublish

Résumé

Primary progressive aphasia (PPA) is mainly considered a sporadic disease and few studies have systematically analyzed its genetic basis. We here report the analyses of C9orf72 genotyping and whole-exome sequencing data in a consecutive and well-characterized cohort of 50 patients with PPA. We identified three pathogenic GRN variants, one of them unreported, and two cases with C9orf72 expansions. In addition, one likely pathogenic variant was found in the SQSTM1 gene. Overall, we found 12%of patients carrying pathogenic or likely pathogenic variants. These results support the genetic role in the pathophysiology of a proportion of patients with PPA.

Identifiants

pubmed: 33612544
pii: JAD201310
doi: 10.3233/JAD-201310
doi:

Substances chimiques

C9orf72 Protein 0
C9orf72 protein, human 0
GRN protein, human 0
Progranulins 0
SQSTM1 protein, human 0
Sequestosome-1 Protein 0

Types de publication

Letter

Langues

eng

Sous-ensembles de citation

IM

Pagination

985-990

Auteurs

Vanesa Pytel (V)

Department of Neurology, Hospital Clinico San Carlos, Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.
Laboratory of Neurobiology, Hospital Clinico San Carlos. Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

Laura Hernández-Lorenzo (L)

Department of Neurology, Hospital Clinico San Carlos, Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.
Laboratory of Neurobiology, Hospital Clinico San Carlos. Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

Laura Torre-Fuentes (L)

Laboratory of Neurobiology, Hospital Clinico San Carlos. Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

Raúl Sanz (R)

Center of Genetic Studies ATG Medical, Madrid, Spain.

Nieves González (N)

Center of Genetic Studies ATG Medical, Madrid, Spain.

María Nieves Cabrera-Martín (MN)

Department of Nuclear Medicine, Hospital Clinico San Carlos, Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

Alfonso Delgado-Álvarez (A)

Department of Neurology, Hospital Clinico San Carlos, Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

Ulises Gómez-Pinedo (U)

Laboratory of Neurobiology, Hospital Clinico San Carlos. Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

Jorge Matías-Guiu (J)

Department of Neurology, Hospital Clinico San Carlos, Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.
Laboratory of Neurobiology, Hospital Clinico San Carlos. Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

Jordi A Matias-Guiu (JA)

Department of Neurology, Hospital Clinico San Carlos, Health Research Institute "San Carlos" (IdISCC), Universidad Complutense de Madrid, Madrid, Spain.

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Classifications MeSH