Fanconi anemia (FA) is a complex tumor-prone disease defined by an entangled genotype and phenotype. Despite enormous efforts in the last 20 years, a comprehensive and integrated view of the disease is still missing. The aim of this pilot study was to establish whether a global microRNA (miRNA) analysis approach could be helpful in defining aspects in FA phenotype, which might deserve future attention with the perspective to develop miRNA-based therapies.
A Global MicroRNA Profile in Fanconi Anemia: A Pilot Study
Degan, Paolo;Longobardi, MariaGrazia;Pulliero, Alessandra;Cuccarolo, Paola;Dufour, Carlo;Ravera, Silvia;Calzia, Daniela;Izzotti, Alberto
2019-01-01
Abstract
Fanconi anemia (FA) is a complex tumor-prone disease defined by an entangled genotype and phenotype. Despite enormous efforts in the last 20 years, a comprehensive and integrated view of the disease is still missing. The aim of this pilot study was to establish whether a global microRNA (miRNA) analysis approach could be helpful in defining aspects in FA phenotype, which might deserve future attention with the perspective to develop miRNA-based therapies.
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