☆ 4.7 Editorial Material

RBC, heal thyself: PK activators in SCD Comment

BLOOD (2022)

A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease

Julia Z. Xu et al.

Summary: This study evaluates the safety and tolerability of mitapivat in SCD patients and finds that it can increase hemoglobin levels and decrease 2,3-DPG concentration, showing potential therapeutic effects for SCD patients.

BLOOD (2022)

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Letter Hematology

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

Minke A. E. Rab et al.

BLOOD (2021)

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Article Biochemical Research Methods

Integrating deep learning with microfluidics for biophysical classification of sickle red blood cells adhered to laminin

Niksa Praljak et al.

Summary: This study introduces an automated system for biophysical characterization of adhered sickle red blood cells in microfluidic devices using deep learning image analysis, showing that the system achieves accuracies matching human experts but with less variance in counts and faster processing time per image. This standardized and reproducible approach holds promise for affordable and high throughput disease monitoring.

PLOS COMPUTATIONAL BIOLOGY (2021)

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Article Physiology

Red Blood Cell Metabolism in Pyruvate Kinase Deficient Patients

Micaela K. Roy et al.

Summary: In the study on PKD patients, metabolomics analyses revealed metabolic disruptions including reduced late glycolysis, accumulation of PPP metabolites, oxidant stress, elevated hypoxia markers, and membrane lipid oxidation and remodeling. This study highlights the importance of basic science research in investigating pathologies in underrepresented minorities and aims to target treatments to health disparities more effectively.

FRONTIERS IN PHYSIOLOGY (2021)

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Review Pharmacology & Pharmacy