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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Vydáno v:Blood
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Hematology 2017
Témata:
Red Cells, Iron, and Erythropoiesis
On-line přístup: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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