Vitamin B5 and succinyl-CoA improve ineffective erythropoiesis in SF3B1 mutated myelodysplasia

Patients with myelodysplastic syndrome and ring sideroblasts (MDS-RS) present with symptomatic anemia due to ineffective erythropoiesis that impede their quality of life and increase morbidity. More than 80% of patients with MDS-RS harbor splicing factor 3B subunit 1 (SF3B1) mutations, the founder a...

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Pubblicato in:Sci Transl Med
Autori principali: Mian, Syed A, Philippe, Céline, Maniati, Eleni, Protopapa, Pantelitsa, Bergot, Tiffany, Piganeau, Marion, Nemkov, Travis, Bella, Doriana Di, Morales, Valle, Finch, Andrew J, D’Alessandro, Angelo, Bianchi, Katiuscia, Wang, Jun, Gallipoli, Paolo, Kordasti, Shahram, Kubasch, Anne Sophie, Cross, Michael, Platzbecker, Uwe, Wiseman, Daniel H, Bonnet, Dominique, Bernard, Delphine G, Gribben, John G, Rouault-Pierre, Kevin
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2023
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7614516/
https://ncbi.nlm.nih.gov/pubmed/36857430
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.abn5135
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