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A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease.
Xu JZ, Conrey A, Frey I, Gwaabe E, Menapace LA, Tumburu L, Lundt M, Lequang T, Li Q, Glass K, Dunkelberger EB, Iyer V, Mangus H, Kung C, Dang L, Kosinski PA, Hawkins P, Jeffries N, Eaton WA, Lay Thein S. Xu JZ, et al. Among authors: li q. Blood. 2022 Nov 10;140(19):2053-2062. doi: 10.1182/blood.2022015403. Blood. 2022. PMID: 35576529 Free PMC article. Clinical Trial.
Kinetic assay shows that increasing red cell volume could be a treatment for sickle cell disease.
Li Q, Henry ER, Hofrichter J, Smith JF, Cellmer T, Dunkelberger EB, Metaferia BB, Jones-Straehle S, Boutom S, Christoph GW, Wakefield TH, Link ME, Staton D, Vass ER, Miller JL, Hsieh MM, Tisdale JF, Eaton WA. Li Q, et al. Proc Natl Acad Sci U S A. 2017 Jan 31;114(5):E689-E696. doi: 10.1073/pnas.1619054114. Epub 2017 Jan 17. Proc Natl Acad Sci U S A. 2017. PMID: 28096387 Free PMC article.
Allosteric control of hemoglobin S fiber formation by oxygen and its relation to the pathophysiology of sickle cell disease.
Henry ER, Cellmer T, Dunkelberger EB, Metaferia B, Hofrichter J, Li Q, Ostrowski D, Ghirlando R, Louis JM, Moutereau S, Galactéros F, Thein SL, Bartolucci P, Eaton WA. Henry ER, et al. Among authors: li q. Proc Natl Acad Sci U S A. 2020 Jun 30;117(26):15018-15027. doi: 10.1073/pnas.1922004117. Epub 2020 Jun 11. Proc Natl Acad Sci U S A. 2020. PMID: 32527859 Free PMC article.
Treatment of sickle cell disease by increasing oxygen affinity of hemoglobin.
Henry ER, Metaferia B, Li Q, Harper J, Best RB, Glass KE, Cellmer T, Dunkelberger EB, Conrey A, Thein SL, Bunn HF, Eaton WA. Henry ER, et al. Among authors: li q. Blood. 2021 Sep 30;138(13):1172-1181. doi: 10.1182/blood.2021012070. Blood. 2021. PMID: 34197597 Free PMC article.
Mitapivat increases ATP and decreases oxidative stress and erythrocyte mitochondria retention in a SCD mouse model.
Quezado ZMN, Kamimura S, Smith M, Wang X, Heaven MR, Jana S, Vogel S, Zerfas P, Combs CA, Almeida LEF, Li Q, Quezado M, Horkayne-Szakaly I, Kosinski PA, Yu S, Kapadnis U, Kung C, Dang L, Wakim P, Eaton WA, Alayash AI, Thein SL. Quezado ZMN, et al. Among authors: li q. Blood Cells Mol Dis. 2022 Jul;95:102660. doi: 10.1016/j.bcmd.2022.102660. Epub 2022 Mar 12. Blood Cells Mol Dis. 2022. PMID: 35366607 Free PMC article.
Phenotypic screening of the ReFRAME drug repurposing library to discover new drugs for treating sickle cell disease.
Metaferia B, Cellmer T, Dunkelberger EB, Li Q, Henry ER, Hofrichter J, Staton D, Hsieh MM, Conrey AK, Tisdale JF, Chatterjee AK, Thein SL, Eaton WA. Metaferia B, et al. Among authors: li q. Proc Natl Acad Sci U S A. 2022 Oct 4;119(40):e2210779119. doi: 10.1073/pnas.2210779119. Epub 2022 Sep 26. Proc Natl Acad Sci U S A. 2022. PMID: 36161945 Free PMC article.
In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation.
Leonard AK, Furstenau D, Inam Z, Luckett C, Chu R, Demirci S, Essawi K, Gudmundsdottir B, Hinds M, DiNicola J, Li Q, Eaton WA, Cellmer T, Wang X, Thein SL, Macari ER, VanNest S, Hsieh MM, Bonner M, Pierciey FJ, Tisdale JF. Leonard AK, et al. Among authors: li q. Blood Adv. 2024 Apr 9;8(7):1806-1816. doi: 10.1182/bloodadvances.2023011397. Blood Adv. 2024. PMID: 38181784 Free PMC article. Clinical Trial.
Functional and multi-omics signatures of mitapivat efficacy upon activation of pyruvate kinase in red blood cells from patients with sickle cell disease.
D'Alessandro A, Le K, Lundt M, Li Q, Dunkelberger EB, Cellmer T, Worth AJ, Patil S, Huston C, Grier A, Dzieciatkowska M, Stephenson D, Eaton WA, Thein SL. D'Alessandro A, et al. Among authors: li q. Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284831. Online ahead of print. Haematologica. 2024. PMID: 38450513 Free article.
βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells.
Demirci S, Gudmundsdottir B, Li Q, Haro-Mora JJ, Nassehi T, Drysdale C, Yapundich M, Gamer J, Seifuddin F, Tisdale JF, Uchida N. Demirci S, et al. Among authors: li q. Mol Ther Methods Clin Dev. 2020 Apr 18;17:912-921. doi: 10.1016/j.omtm.2020.04.013. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32405513 Free PMC article.
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