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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Detalhes bibliográficos
Publicado no:PLoS Med
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2017
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Research Article
Acesso em linha: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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