Voxelotor: alteration of sickle cell disease pathophysiology by a first-in-class polymerization inhibitor

AK Glaros, R Razvi, N Shah… - Therapeutic advances in …, 2021 - journals.sagepub.com
Sickle cell disease, despite its recognition as a severely debilitating genetic condition
affecting hundreds of thousands of neonates throughout the world each year, was not a …

Genetic variants of PKLR are associated with acute pain in sickle cell disease

X Wang, K Gardner, MB Tegegn, CL Dalgard… - Blood …, 2022 - ashpublications.org
Acute pain, the most prominent complication of sickle cell disease (SCD), results from vaso-
occlusion triggered by sickling of deoxygenated red blood cells (RBCs). Concentration of 2 …

An integrated therapeutic approach to sickle cell disease management beyond infancy

JA Ribeil, G Pollock, H Frangoul… - American Journal of …, 2023 - Wiley Online Library
Hydroxyurea, the first approved drug for sickle cell disease, decreases sickle hemoglobin
polymerization by inducing fetal hemoglobin. Its effects in young children are excellent; …

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

ZMN Quezado, S Kamimura, M Smith, X Wang… - Blood Cells, Molecules …, 2022 - Elsevier
Polymerization of deoxygenated sickle hemoglobin (HbS) leads to erythrocyte sickling.
Enhancing activity of the erythrocyte glycolytic pathway has anti-sickling potential as this …

[HTML][HTML] Fatty acid nitroalkenes–Multi-target agents for the treatment of sickle cell disease

FA Chowdhury, N Colussi, M Sharma, KC Wood, JZ Xu… - Redox Biology, 2023 - Elsevier
Sickle cell disease (SCD) is a hereditary hematological disease with high morbidity and
mortality rates worldwide. Despite being monogenic, SCD patients display a plethora of …

Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations

KJ Woodard, PA Doerfler… - Disease Models & …, 2022 - journals.biologists.com
We characterized the human β-like globin transgenes in two mouse models of sickle cell
disease (SCD) and tested a genome-editing strategy to induce red blood cell fetal …

Outcomes of pregnancy in sickle cell disease patients: results from the prospective ESCORT-HU cohort study

A Habibi, G Cannas, P Bartolucci, E Voskaridou… - Biomedicines, 2023 - mdpi.com
Sickle cell disease (SCD) refers to a group of inherited hemoglobin disorders in which sickle
red blood cells display altered deformability, leading to a significant burden of acute and …

PIEZO1 and the mechanism of the long circulatory longevity of human red blood cells

S Rogers, VL Lew - PLoS Computational Biology, 2021 - journals.plos.org
Human red blood cells (RBCs) have a circulatory lifespan of about four months. Under
constant oxidative and mechanical stress, but devoid of organelles and deprived of …

Development of pathophysiologically relevant models of sickle cell disease and β-thalassemia for therapeutic studies

P Gupta, SG Goswami, G Kumari… - Nature …, 2024 - nature.com
Ex vivo cellular system that accurately replicates sickle cell disease and β-thalassemia
characteristics is a highly sought-after goal in the field of erythroid biology. In this study, we …

How I treat the older adult with sickle cell disease

SL Thein, J Howard - Blood, The Journal of the American …, 2018 - ashpublications.org
With increasing survival, cumulative complications of sickle cell disease (SCD), which
develop insidiously over time, are becoming more apparent and common in older patients …