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Multiomic Approaches to Uncover the Complexities of Dystrophin-Associated Cardiomyopathy

Despite major progress in treating skeletal muscle disease associated with dystrophinopathies, cardiomyopathy is emerging as a major cause of death in people carrying dystrophin gene mutations that remain without a targeted cure even with new treatment directions and advances in modelling abilities....

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Detalles Bibliográficos
Autores principales: Gowran, Aoife, Brioschi, Maura, Rovina, Davide, Chiesa, Mattia, Piacentini, Luca, Mallia, Sara, Banfi, Cristina, Pompilio, Giulio, Santoro, Rosaria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8396646/
https://www.ncbi.nlm.nih.gov/pubmed/34445659
http://dx.doi.org/10.3390/ijms22168954
Descripción
Sumario:Despite major progress in treating skeletal muscle disease associated with dystrophinopathies, cardiomyopathy is emerging as a major cause of death in people carrying dystrophin gene mutations that remain without a targeted cure even with new treatment directions and advances in modelling abilities. The reasons for the stunted progress in ameliorating dystrophin-associated cardiomyopathy (DAC) can be explained by the difficulties in detecting pathophysiological mechanisms which can also be efficiently targeted within the heart in the widest patient population. New perspectives are clearly required to effectively address the unanswered questions concerning the identification of authentic and effectual readouts of DAC occurrence and severity. A potential way forward to achieve further therapy breakthroughs lies in combining multiomic analysis with advanced preclinical precision models. This review presents the fundamental discoveries made using relevant models of DAC and how omics approaches have been incorporated to date.