Mutation update for the ACTN2 gene

ACTN2 encodes alpha-actinin-2, a protein expressed in human cardiac and skeletal muscle. The protein, located in the sarcomere Z-disk, functions as a link between the anti-parallel actin filaments. This important structural protein also binds N-terminal titins, and thus contributes to sarcomere stab...

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Detalles Bibliográficos
Autores: Ranta-aho, Johanna, Olivé i Plana, Montserrat|||0000-0001-5727-0165, Vandroux, Marie, Roticiani, Giorgia, Domínguez-González, Cristina|||0000-0001-5151-988X, Johari, Mridul|||0000-0002-3549-558X, Torella, Annalaura, Böhm, Johann|||0000-0001-8019-9504, Turon-Sans, Janina|||0000-0002-8842-4646, Nigro, Vincenzo, Hackman, Peter, Laporte, Jocelyn, Udd, Bjarne, Savarese, Marco|||0000-0002-2591-244X
Tipo de recurso: artículo
Fecha de publicación:2022
País:España
Institución:Universitat Autònoma de Barcelona
Repositorio:Dipòsit Digital de Documents de la UAB
Idioma:inglés
OAI Identifier:oai:ddd.uab.cat:286324
Acceso en línea:https://ddd.uab.cat/record/286324
https://dx.doi.org/urn:doi:10.1002/humu.24470
Access Level:acceso abierto
Palabra clave:ACTN2
Alpha-actinin-2
Cardiomyopathy
Congenital myopathy
Distal myopathy
Descripción
Sumario:ACTN2 encodes alpha-actinin-2, a protein expressed in human cardiac and skeletal muscle. The protein, located in the sarcomere Z-disk, functions as a link between the anti-parallel actin filaments. This important structural protein also binds N-terminal titins, and thus contributes to sarcomere stability. Previously, ACTN2 mutations have been solely associated with cardiomyopathy, without skeletal muscle disease. Recently, however, ACTN2 mutations have been associated with novel congenital and distal myopathy. Previously reported variants are in varying locations across the gene, but the potential clustering effect of pathogenic locations is not clearly understood. Further, the genotype-phenotype correlations of these variants remain unclear. Here we review the previously reported ACTN2-related molecular and clinical findings and present an additional variant, c.1840-2A.