Targeted sequencing of selected functional genes in patients with wild-type transthyretin amyloidosis

[Objective]: Wild-type transthyretin (ATTRwt) amyloidosis is caused by the misfolding and deposition of the transthyretin protein (TTR) in the absence of mutations in the TTR gene. Studies regarding the variant form of ATTR amyloidosis (ATTRv) suggest that the presence of single-nucleotide polymorph...

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Detalles Bibliográficos
Autores: Moreno-Gázquez, Inmaculada, Pérez-Palacios, Raquel, Abengochea-Quílez, Lucia, Lahuerta Pueyo, Carmen, Roteta Unceta Barrenechea, Ana, Andrés Gracia, Alejandro, Aibar Arregui, Miguel Angel, Menao Guillén, Sebastián
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2023
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/350708
Acceso en línea:http://hdl.handle.net/10261/350708
Access Level:acceso abierto
Palabra clave:ATTRwt amyloidosis
Transthyretin amyloidosis
Gene variants
Targeted sequencing
Descripción
Sumario:[Objective]: Wild-type transthyretin (ATTRwt) amyloidosis is caused by the misfolding and deposition of the transthyretin protein (TTR) in the absence of mutations in the TTR gene. Studies regarding the variant form of ATTR amyloidosis (ATTRv) suggest that the presence of single-nucleotide polymorphisms (SNP) in genes other than the TTR, may influence the development of the disease. However, other genetic factors involved in the aetiopathogenesis of ATTRwt are currently unknown. This work investigates the presence of sequence variants in genes selected for their possible impact on ATTRwt amyloidosis. To do so, targeted sequencing of 84 protein-coding genes was performed in a cohort of 27 patients diagnosed with ATTRwt.