AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency

Pyruvate kinase (PK) deficiency is a rare genetic disease that causes chronic hemolytic anemia. There are currently no targeted therapies for PK deficiency. Here, we describe the identification and characterization of AG-348, an allosteric activator of PK that is currently in clinical trials for the...

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Detalhes bibliográficos
Publicado no:Blood
Main Authors: Kung, Charles, Hixon, Jeff, Kosinski, Penelope A., Cianchetta, Giovanni, Histen, Gavin, Chen, Yue, Hill, Collin, Gross, Stefan, Si, Yaguang, Johnson, Kendall, DeLaBarre, Byron, Luo, Zhiyong, Gu, Zhiwei, Yao, Gui, Tang, Huachun, Fang, Cheng, Xu, Yingxia, Lv, Xiaobing, Biller, Scott, Su, Shin-San Michael, Yang, Hua, Popovici-Muller, Janeta, Salituro, Francesco, Silverman, Lee, Dang, Lenny
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2017
Assuntos:
Red Cells, Iron, and Erythropoiesis
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5609468/
https://ncbi.nlm.nih.gov/pubmed/28760888
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2016-11-753525
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