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Emerging Therapeutic Approaches for Diamond Blackfan Anemia

Diamond Blackfan Anemia (DBA) is an inherited erythroid aplasia with onset in childhood. Patients carry heterozygous mutations in one of 19 Ribosomal Protein (RP) genes, that lead to defective ribosome biogenesis and function. Standard treatments include steroids or blood transfusions but the only d...

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Dades bibliogràfiques
Publicat a:Curr Gene Ther
Autors principals: Aspesi, Anna, Borsotti, Chiara, Follenzi, Antonia
Format: Artigo
Idioma:Inglês
Publicat: Bentham Science Publishers 2018
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6637096/
https://ncbi.nlm.nih.gov/pubmed/30411682
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2174/1566523218666181109124538
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