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Digenic inheritance of STUB1variants and TBPpolyglutamine expansions explains the incomplete penetrance of SCA17 and SCA48

Authors :
Magri, Stefania
Nanetti, Lorenzo
Gellera, Cinzia
Sarto, Elisa
Rizzo, Elena
Mongelli, Alessia
Ricci, Benedetta
Fancellu, Roberto
Sambati, Luisa
Cortelli, Pietro
Brusco, Alfredo
Bruzzone, Maria Grazia
Mariotti, Caterina
Di Bella, Daniela
Taroni, Franco
Source :
Genetics in Medicine; January 2022, Vol. 24 Issue: 1 p29-40, 12p
Publication Year :
2022

Abstract

This study aimed to unravel the genetic factors underlying missing heritability in spinocerebellar ataxia type 17 (SCA17) caused by polyglutamine-encoding CAG/CAA repeat expansions in the TBPgene. Alleles with >49 CAG/CAA repeats are fully penetrant. Most patients, however, carry intermediate TBP41-49alleles that show incomplete penetrance.

Details

Language :
English
ISSN :
10983600 and 15300366
Volume :
24
Issue :
1
Database :
Supplemental Index
Journal :
Genetics in Medicine
Publication Type :
Periodical
Accession number :
ejs58800167
Full Text :
https://doi.org/10.1016/j.gim.2021.08.003