Title : Azacitidine is a potential therapeutic drug for pyridoxine-refractory female X-linked sideroblastic anemia.

Pub. Date : 2022 Feb 22

PMID : 34781359






2 Functional Relationships(s)
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1 Administration of a DNA demethylating agent, azacitidine, reactivated the silent wild-type ALAS2 allele in active mutant HPCs and ameliorated erythroid differentiation defects, suggesting that azacitidine is a potential novel therapeutic drug for female XLSA. Azacitidine 5'-aminolevulinate synthase 2 Homo sapiens
2 Administration of a DNA demethylating agent, azacitidine, reactivated the silent wild-type ALAS2 allele in active mutant HPCs and ameliorated erythroid differentiation defects, suggesting that azacitidine is a potential novel therapeutic drug for female XLSA. Azacitidine 5'-aminolevulinate synthase 2 Homo sapiens