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RAP-011 Rescues the Disease Phenotype in a Cellular Model of Congenital Dyserythropoietic Anemia Type II by Inhibiting the SMAD2-3 Pathway

Congenital dyserythropoietic anemia type II (CDA II) is a hypo-productive anemia defined by ineffective erythropoiesis through maturation arrest of erythroid precursors. CDA II is an autosomal recessive disorder due to loss-of-function mutations in SEC23B. Currently, management of patients with CDA...

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Detalles Bibliográficos
Autores principales: De Rosa, Gianluca, Andolfo, Immacolata, Marra, Roberta, Manna, Francesco, Rosato, Barbara Eleni, Iolascon, Achille, Russo, Roberta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7432210/
https://www.ncbi.nlm.nih.gov/pubmed/32759740
http://dx.doi.org/10.3390/ijms21155577

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