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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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Bibliografski detalji
Izdano u:Curr Gene Ther
Glavni autori: Aspesi, Anna, Borsotti, Chiara, Follenzi, Antonia
Format: Artigo
Jezik:Inglês
Izdano: Bentham Science Publishers 2018
Teme:
Online pristup: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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