Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study

BACKGROUND: Sickle cell disease (SCD), a congenital hemolytic anemia that exacts terrible global morbidity and mortality, is driven by polymerization of mutated sickle hemoglobin (HbS) in red blood cells (RBCs). Fetal hemoglobin (HbF) interferes with this polymerization, but HbF is epigenetically si...

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Bibliografski detalji
Izdano u:PLoS Med
Glavni autori: Molokie, Robert, Lavelle, Donald, Gowhari, Michel, Pacini, Michael, Krauz, Lani, Hassan, Johara, Ibanez, Vinzon, Ruiz, Maria A., Ng, Kwok Peng, Woost, Philip, Radivoyevitch, Tomas, Pacelli, Daisy, Fada, Sherry, Rump, Matthew, Hsieh, Matthew, Tisdale, John F., Jacobberger, James, Phelps, Mitch, Engel, James Douglas, Saraf, Santhosh, Hsu, Lewis L., Gordeuk, Victor, DeSimone, Joseph, Saunthararajah, Yogen
Format: Artigo
Jezik:Inglês
Izdano: Public Library of Science 2017
Teme:
Research Article
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5589090/
https://ncbi.nlm.nih.gov/pubmed/28880867
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pmed.1002382
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