Deciphering autism spectrum disorder genomic variation through the characterization of zebrafish and human stem cell-based models

Autism Spectrum Disorders are characterized by behavioural, social, and communication impairments. Despite their prevalence, the precise mechanisms underlying ASD are poorly understood. This thesis is focused on developing suitable models to study the impact of mutations in three genes (BTBD8, FOSL2...

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Detalles Bibliográficos
Autor: Veiga Rúa, Sara
Tipo de recurso: tesis doctoral
Fecha de publicación:2024
País:España
Institución:Universidad de Santiago de Compostela (USC)
Repositorio:Minerva. Repositorio Institucional de la Universidad de Santiago de Compostela
Idioma:inglés
OAI Identifier:oai:minerva.usc.gal:10347/33975
Acceso en línea:http://hdl.handle.net/10347/33975
Access Level:acceso abierto
Palabra clave:240902 Ingeniería genética
Descripción
Sumario:Autism Spectrum Disorders are characterized by behavioural, social, and communication impairments. Despite their prevalence, the precise mechanisms underlying ASD are poorly understood. This thesis is focused on developing suitable models to study the impact of mutations in three genes (BTBD8, FOSL2, and TAOK1) potentially associated with ASD. In vitro models were generated using human iPSCs that were differentiated into cortical neurons and human brain organoids, that were characterized through proteomics and scRNA-seq. Genes of interest were also studied in Danio rerio, to evaluate the morphological and behavioural consequences of gene dysfunction. These models will be useful for improving our understanding of the molecular mechanisms underlying ASD, but also for improving the diagnosis and treatment of the disorder.