Systematic Screening of Ubiquitin/p62 Aggregates in Cerebellar Cortex Expands the Neuropathological Phenotype of the C9orf72 Expansion Mutation


Por: Ramos-Campoy, O, Avila-Polo, R, Grau-Rivera, O, Antonell, A, Clarimon, J, Rojas-Garcia, R, Charif, S, Santiago-Valera, V, Hernandez, I, Aguilar, M, Almenar, C, Lopez-Villegas, D, Bajo, L, Pastor, P, Van der Zee, J, Llado, A, Sanchez-Valle, R, Gelpi, E

Publicada: 1 ago 2018
Resumen:
The neuropathological hallmark of the C9orf72 intronic hexanucleotide expansion in frontotemporal lobar degeneration (FTLD) and amyotrophic lateral sclerosis (ALS) is the presence of small ubiquitin/p62-positive and transactive response DNA binding protein 43 kDa (TDP-43)-negative cytoplasmic inclusions in several brain areas. The identification of this histopathological signature is highly predictive of an underlying mutation. In this study, we screened 1800 cases of the Barcelona IDIBAPS Brain Bank, independently of the clinical and final neuropathological diagnosis of the brain donor, for the presence of ubiquitin/p62-positive inclusions in the cerebellum (UPPI). Positive cases were also stained for dipeptide repeats. We identified a total of 21 donors with UPPI and in all of them the C9orf72 hexanucleotide expansion was genetically confirmed. Most donors had an FTLD or to a lesser extent ALS clinico-pathological phenotype. However, 3 cases had been previously classified as having clinically and neuropathologically Lewy body disease. Other co-existing pathologies, especially of the PART-type, were also frequently encountered. This study highlights the importance of the evaluation of ubiquitin/p62-positive cytoplasmic inclusions in all neurodegenerative diseases as a good screening method for the detection of C9orf72 expansion mutation, since this mutation is not rare and can overlap with other neurodegenerative entities.

Filiaciones:
Ramos-Campoy, O:
 Hosp Clin Barcelona, Neurol Tissue Bank, IDI, Biobanc,BAPS, Barcelona, Spain

 Hosp Clin Barcelona, Alzheimer Dis & Other Cognit Disorders Unit, Dept Neurol, Barcelona, Spain

Avila-Polo, R:
 Hosp Clin Barcelona, Neurol Tissue Bank, IDI, Biobanc,BAPS, Barcelona, Spain

 Hosp Univ Virgen del Rocio, Dept Anat Pathol, FISEVI, Seville, Spain

Grau-Rivera, O:
 Hosp Clin Barcelona, Neurol Tissue Bank, IDI, Biobanc,BAPS, Barcelona, Spain

 Hosp Clin Barcelona, Alzheimer Dis & Other Cognit Disorders Unit, Dept Neurol, Barcelona, Spain

Antonell, A:
 Hosp Clin Barcelona, Alzheimer Dis & Other Cognit Disorders Unit, Dept Neurol, Barcelona, Spain

Clarimon, J:
 Univ Autonoma Barcelona, Dept Neurol, Hosp Santa Creu & St Pau, Barcelona, Spain

 Univ Autonoma Barcelona, St Pau Biomed Res Inst, Hosp Santa Creu & St Pau, Barcelona, Spain

 Univ Autonoma Barcelona, Hosp Santa Creu & St Pau, Ctr Networked Biomed Res Neurodegenerat Dis CIBER, Neuromuscular Dis Unit,Dept Neurol, Barcelona, Spain

Rojas-Garcia, R:
 Ctr Networked Biomed Res Rare Dis CIBERER, Barcelona, Spain

Charif, S:
 Hosp Clin Barcelona, Neurol Tissue Bank, IDI, Biobanc,BAPS, Barcelona, Spain

Santiago-Valera, V:
 Hosp Clin Barcelona, Neurol Tissue Bank, IDI, Biobanc,BAPS, Barcelona, Spain

Hernandez, I:
 Fdn ACE, Barcelona, Spain

Aguilar, M:
 Univ Hosp Mutua Terrassa, Movement Disorders & Memory Unit, Dept Neurol, Barcelona, Spain

 Fdn Recerca Biomed & Social Mutua Terrassa, Barcelona, Spain

Almenar, C:
 Hosp Germanes Hosp Benito Menni, Dept Neurol, St Boi de Llobregat, Barcelona, Spain

Lopez-Villegas, D:
 Dept Neurol, Parc Salut Mar, Barcelona, Spain

Bajo, L:
 Hosp Univ Santa Creu de Vic, Dept Geriatry, Barcelona, Spain

Pastor, P:
 Univ Hosp Mutua Terrassa, Movement Disorders & Memory Unit, Dept Neurol, Barcelona, Spain

 Fdn Recerca Biomed & Social Mutua Terrassa, Barcelona, Spain

Van der Zee, J:
 Univ Antwerp, Inst Born Bunge, Antwerp, Belgium

Llado, A:
 Hosp Clin Barcelona, Alzheimer Dis & Other Cognit Disorders Unit, Dept Neurol, Barcelona, Spain

Sanchez-Valle, R:
 Hosp Clin Barcelona, Neurol Tissue Bank, IDI, Biobanc,BAPS, Barcelona, Spain

 Hosp Clin Barcelona, Alzheimer Dis & Other Cognit Disorders Unit, Dept Neurol, Barcelona, Spain

Gelpi, E:
 Hosp Clin Barcelona, Neurol Tissue Bank, IDI, Biobanc,BAPS, Barcelona, Spain

 Med Univ Vienna, Inst Neurol, Waehringer Guertel 18-20, A-1097 Vienna, Austria
ISSN: 00223069





JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY
Editorial
OXFORD UNIV PRESS INC, JOURNALS DEPT, 2001 EVANS RD, CARY, NC 27513 USA, USA
Tipo de documento: Article
Volumen: 77 Número: 8
Páginas: 703-709
WOS Id: 000440966500006
ID de PubMed: 29889265
imagen Bronze, Green Accepted

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