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Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion.

Authors :
Campuzano, V
Montermini, L
Moltò, M D
Pianese, Luigi
Cossée, Mireille
Cavalcanti, F
Monros, E
Rodius, F
Duclos, F
Monticelli, Antonella
Zara, F
Cañizares, J
Koutnikova, H
Bidichandani, S I
Gellera, Cinzia
Brice, Alexis
Trouillas, P
De Michele, G
Filla, A
de Frutos, R
Palau, F
Patel, P I
di Donato, S
Mandel, J L
Cocozza, Sergio
Koenig, Michel
Pandolfo, Massimo
Campuzano, V
Montermini, L
Moltò, M D
Pianese, Luigi
Cossée, Mireille
Cavalcanti, F
Monros, E
Rodius, F
Duclos, F
Monticelli, Antonella
Zara, F
Cañizares, J
Koutnikova, H
Bidichandani, S I
Gellera, Cinzia
Brice, Alexis
Trouillas, P
De Michele, G
Filla, A
de Frutos, R
Palau, F
Patel, P I
di Donato, S
Mandel, J L
Cocozza, Sergio
Koenig, Michel
Pandolfo, Massimo
Source :
Science, 271 (5254
Publication Year :
1996

Abstract

Friedreich's ataxia (FRDA) is an autosomal recessive, degenerative disease that involves the central and peripheral nervous systems and the heart. A gene, X25, was identified in the critical region for the FRDA locus on chromosome 9q13. This gene encodes a 210-amino acid protein, frataxin, that has homologs in distant species such as Caenorhabditis elegans and yeast. A few FRDA patients were found to have point mutations in X25, but the majority were homozygous for an unstable GAA trinucleotide expansion in the first X25 intron.<br />Journal Article<br />Research Support, Non-U.S. Gov't<br />Research Support, U.S. Gov't, P.H.S.<br />info:eu-repo/semantics/published

Details

Database :
OAIster
Journal :
Science, 271 (5254
Notes :
No full-text files, English
Publication Type :
Electronic Resource
Accession number :
edsoai.on1363714877
Document Type :
Electronic Resource