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


In a clinical trial, Yogen Saunthararajah and colleagues target DNA methyltransferase with small molecules to reactivate fetal hemoglobin which inhibits polymerisation of mutated sickle cell hemoglobin.


Vyšlo v časopise: Oral tetrahydrouridine and decitabine for non-cytotoxic epigenetic gene regulation in sickle cell disease: A randomized phase 1 study. PLoS Med 14(9): e32767. doi:10.1371/journal.pmed.1002382
Kategorie: Research Article
prolekare.web.journal.doi_sk: https://doi.org/10.1371/journal.pmed.1002382

Souhrn

In a clinical trial, Yogen Saunthararajah and colleagues target DNA methyltransferase with small molecules to reactivate fetal hemoglobin which inhibits polymerisation of mutated sickle cell hemoglobin.


Zdroje

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