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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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Foilsithe in:Curr Gene Ther
Main Authors: Aspesi, Anna, Borsotti, Chiara, Follenzi, Antonia
Formáid: Artigo
Teanga:Inglês
Foilsithe: Bentham Science Publishers 2018
Ábhair:
Rochtain Ar Líne: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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