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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5589090/
https://www.ncbi.nlm.nih.gov/pubmed/28880867
http://dx.doi.org/10.1371/journal.pmed.1002382

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