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Lista de obras de Mattia Ferrarese

Akt-mediated phosphorylation of MICU1 regulates mitochondrial Ca2+ levels and tumor growth

artículo científico publicado en 2018

An engineered human albumin enhances half-life and transmucosal delivery when fused to protein-based biologics

artículo científico publicado en 2020

Differential functional readthrough over homozygous nonsense mutations contributes to the bleeding phenotype in coagulation factor VII deficiency.

artículo científico publicado en 2016

Missense changes in the catalytic domain of coagulation factor X account for minimal function preventing a perinatal lethal condition

artículo científico publicado en 2019

Secretion of wild-type factor IX upon readthrough over F9 pre-peptide nonsense mutations causing hemophilia B.

artículo científico publicado en 2018

Specific factor IX mRNA and protein features favor drug-induced readthrough over recurrent nonsense mutations

artículo científico publicado en 2017

The carboxyl-terminal region of human coagulation factor X as a natural linker for fusion strategies

scientific article published on 09 November 2018

The chaperone-like sodium phenylbutyrate improves factor IX intracellular trafficking and activity impaired by the frequent p.R294Q mutation

artículo científico publicado en 2018

Translational readthrough of GLA nonsense mutations suggests dominant-negative effects exerted by the interaction of wild-type and missense variants

scientific article published on 15 October 2019

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

artículo científico publicado en 2016

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