Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias: Shedding new light on an old enzyme
Publikation: Bidrag til tidsskrift › Review › Forskning › fagfællebedømt
Standard
Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias : Shedding new light on an old enzyme. / van Dijk, Myrthe J.; de Wilde, Jonathan R.A.; Bartels, Marije; Kuo, Kevin H.M.; Glenthøj, Andreas; Rab, Minke A.E.; van Beers, Eduard J.; van Wijk, Richard.
I: Blood Reviews, Bind 61, 101103, 2023.Publikation: Bidrag til tidsskrift › Review › Forskning › fagfællebedømt
Harvard
APA
Vancouver
Author
Bibtex
}
RIS
TY - JOUR
T1 - Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias
T2 - Shedding new light on an old enzyme
AU - van Dijk, Myrthe J.
AU - de Wilde, Jonathan R.A.
AU - Bartels, Marije
AU - Kuo, Kevin H.M.
AU - Glenthøj, Andreas
AU - Rab, Minke A.E.
AU - van Beers, Eduard J.
AU - van Wijk, Richard
N1 - Corrigendum: https://doi.org/10.1016/j.blre.2023.101160 Publisher Copyright: © 2023
PY - 2023
Y1 - 2023
N2 - Novel developments in therapies for various hereditary hemolytic anemias reflect the pivotal role of pyruvate kinase (PK), a key enzyme of glycolysis, in red blood cell (RBC) health. Without PK catalyzing one of the final steps of the Embden-Meyerhof pathway, there is no net yield of adenosine triphosphate (ATP) during glycolysis, the sole source of energy production required for proper RBC function and survival. In hereditary hemolytic anemias, RBC health is compromised and therefore lifespan is shortened. Although our knowledge on glycolysis in general and PK function in particular is solid, recent advances in genetic, molecular, biochemical, and metabolic aspects of hereditary anemias have improved our understanding of these diseases. These advances provide a rationale for targeting PK as therapeutic option in hereditary hemolytic anemias other than PK deficiency. This review summarizes the knowledge, rationale, (pre)clinical trials, and future advances of PK activators for this important group of rare diseases.
AB - Novel developments in therapies for various hereditary hemolytic anemias reflect the pivotal role of pyruvate kinase (PK), a key enzyme of glycolysis, in red blood cell (RBC) health. Without PK catalyzing one of the final steps of the Embden-Meyerhof pathway, there is no net yield of adenosine triphosphate (ATP) during glycolysis, the sole source of energy production required for proper RBC function and survival. In hereditary hemolytic anemias, RBC health is compromised and therefore lifespan is shortened. Although our knowledge on glycolysis in general and PK function in particular is solid, recent advances in genetic, molecular, biochemical, and metabolic aspects of hereditary anemias have improved our understanding of these diseases. These advances provide a rationale for targeting PK as therapeutic option in hereditary hemolytic anemias other than PK deficiency. This review summarizes the knowledge, rationale, (pre)clinical trials, and future advances of PK activators for this important group of rare diseases.
KW - Glycolysis
KW - Hemolytic anemia
KW - Pyruvate kinase
KW - Treatment
U2 - 10.1016/j.blre.2023.101103
DO - 10.1016/j.blre.2023.101103
M3 - Review
C2 - 37353463
AN - SCOPUS:85166909129
VL - 61
JO - Blood Reviews
JF - Blood Reviews
SN - 0268-960X
M1 - 101103
ER -
ID: 388025194