Literature DB >> 33690814

Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy.

Minke A E Rab1,2, Jennifer Bos1, Brigitte A van Oirschot1, Stephanie van Straaten3, Penelope A Kosinski4, Victor Chubukov4, Hyeryun Kim4, Heidi Mangus4, Roger E G Schutgens2, Gerard Pasterkamp1, Lenny Dang4, Charles Kung4, Eduard J van Beers2, Richard van Wijk1.   

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Year:  2021        PMID： 33690814      PMCID： PMC8160499          DOI： 10.1182/blood.2020008635

Source DB:  PubMed          Journal:  Blood        ISSN： 0006-4971            Impact factor:   22.113


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  19 in total

Review 1.  Sickle Cell Disease.

Authors:  Frédéric B Piel; Martin H Steinberg; David C Rees
Journal:  N Engl J Med       Date:  2017-04-20       Impact factor: 91.245

2.  Phase 1 Single- and Multiple-Ascending-Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers.

Authors:  Hua Yang; Elizabeth Merica; Yue Chen; Marvin Cohen; Ronald Goldwater; Penelope A Kosinski; Charles Kung; Zheng Jason Yuan; Lee Silverman; Meredith Goldwasser; Bruce A Silver; Sam Agresta; Ann J Barbier
Journal:  Clin Pharmacol Drug Dev       Date:  2018-08-09

3.  Inhibition of pyruvate kinase M2 by reactive oxygen species contributes to cellular antioxidant responses.

Authors:  Dimitrios Anastasiou; George Poulogiannis; John M Asara; Matthew B Boxer; Jian-kang Jiang; Min Shen; Gary Bellinger; Atsuo T Sasaki; Jason W Locasale; Douglas S Auld; Craig J Thomas; Matthew G Vander Heiden; Lewis C Cantley
Journal:  Science       Date:  2011-11-03       Impact factor: 47.728

4.  Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.

Authors:  Rachael F Grace; Christian Rose; D Mark Layton; Frédéric Galactéros; Wilma Barcellini; D Holmes Morton; Eduard J van Beers; Hassan Yaish; Yaddanapudi Ravindranath; Kevin H M Kuo; Sujit Sheth; Janet L Kwiatkowski; Ann J Barbier; Susan Bodie; Bruce Silver; Lei Hua; Charles Kung; Peter Hawkins; Marie-Hélène Jouvin; Chris Bowden; Bertil Glader
Journal:  N Engl J Med       Date:  2019-09-05       Impact factor: 91.245

5.  The role of red cell energy metabolism in the generation of irreversibly sickled cells in vitro.

Authors:  M Jensen; S B Shohet; D G Nathan
Journal:  Blood       Date:  1973-12       Impact factor: 22.113

6.  Sickle cell disease in a carrier with pyruvate kinase deficiency.

Authors:  Nazeer Alli; Marius Coetzee; Vernon Louw; Ben van Rensburg; Gerrit Rossouw; Lisa Thompson; Serge Pissard; Swee Lay Thein
Journal:  Hematology       Date:  2008-12       Impact factor: 2.269

7.  Reactive oxygen species and phosphatidylserine externalization in murine sickle red cells.

Authors:  Tinku Banerjee; Frans A Kuypers
Journal:  Br J Haematol       Date:  2004-02       Impact factor: 6.998

Review 8.  Erythrocyte pyruvate kinase deficiency: 2015 status report.

Authors:  Rachael F Grace; Alberto Zanella; Ellis J Neufeld; D Holmes Morton; Stefan Eber; Hassan Yaish; Bertil Glader
Journal:  Am J Hematol       Date:  2015-08-14       Impact factor: 10.047

9.  Rapid and reproducible characterization of sickling during automated deoxygenation in sickle cell disease patients.

Authors:  Minke A E Rab; Brigitte A van Oirschot; Jennifer Bos; Tesy H Merkx; Annet C W van Wesel; Osheiza Abdulmalik; Martin K Safo; Birgitta A Versluijs; Maite E Houwing; Marjon H Cnossen; Jurgen Riedl; Roger E G Schutgens; Gerard Pasterkamp; Marije Bartels; Eduard J van Beers; Richard van Wijk
Journal:  Am J Hematol       Date:  2019-03-08       Impact factor: 10.047

10.  AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes.

Authors:  Minke A E Rab; Brigitte A Van Oirschot; Penelope A Kosinski; Jeffrey Hixon; Kendall Johnson; Victor Chubukov; Lenny Dang; Gerard Pasterkamp; Stephanie Van Straaten; Wouter W Van Solinge; Eduard J Van Beers; Charles Kung; Richard Van Wijk
Journal:  Haematologica       Date:  2021-01-01       Impact factor: 9.941
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  3 in total

1.  Revisiting anemia in sickle cell disease and finding the balance with therapeutic approaches.

Authors:  Julia Zhe Xu; Swee Lay Thein
Journal:  Blood       Date:  2022-05-19       Impact factor: 25.476

2.  Mitapivat increases ATP and decreases oxidative stress and erythrocyte mitochondria retention in a SCD mouse model.

Authors:  Zenaide M N Quezado; Sayuri Kamimura; Meghann Smith; Xunde Wang; Michael R Heaven; Sirsendu Jana; Sebastian Vogel; Patricia Zerfas; Christian A Combs; Luis E F Almeida; Quan Li; Martha Quezado; Iren Horkayne-Szakaly; Penelope A Kosinski; Shaoxia Yu; Unnati Kapadnis; Charles Kung; Lenny Dang; Paul Wakim; William A Eaton; Abdu I Alayash; Swee Lay Thein
Journal:  Blood Cells Mol Dis       Date:  2022-03-12       Impact factor: 2.372

Review 3.  Mitapivat, a novel pyruvate kinase activator, for the treatment of hereditary hemolytic anemias.

Authors:  Hanny Al-Samkari; Eduard J van Beers
Journal:  Ther Adv Hematol       Date:  2021-12-21
  3 in total

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