Treatment of sickle cell disease by increasing oxygen affinity of hemoglobin

ER Henry, B Metaferia, Q Li, J Harper… - Blood, The Journal …, 2021 - ashpublications.org
The issue of treating sickle cell disease with drugs that increase hemoglobin oxygen affinity
has come to the fore with the US Food and Drug Administration approval in 2019 of …

Genetic therapies for the first molecular disease

PA Doerfler, A Sharma, JS Porter… - The Journal of …, 2021 - Am Soc Clin Investig
Sickle cell disease (SCD) is a monogenic disorder characterized by recurrent episodes of
severe bone pain, multi-organ failure, and early mortality. Although medical progress over …

Biosynthesis of High‐Active Hemoproteins by the Efficient Heme‐Supply Pichia Pastoris Chassis

F Yu, X Zhao, J Zhou, W Lu, J Li, J Chen… - Advanced …, 2023 - Wiley Online Library
Microbial synthesis of valuable hemoproteins has become a popular research topic, and
Pichia pastoris is a versatile platform for the industrial production of recombinant proteins …

Targeting fetal hemoglobin expression to treat β hemoglobinopathies

MH Steinberg - Expert Opinion on Therapeutic Targets, 2022 - Taylor & Francis
Introduction Sickle cell disease and β thalassemia are the principal β hemoglobinopathies.
The complex pathophysiology of sickle cell disease is initiated by sickle hemoglobin …

Phenotypic screening of the ReFRAME drug repurposing library to discover new drugs for treating sickle cell disease

B Metaferia, T Cellmer… - Proceedings of the …, 2022 - National Acad Sciences
Stem cell transplantation and genetic therapies offer potential cures for patients with sickle
cell disease (SCD), but these options require advanced medical facilities and are expensive …

Feature tracking microfluidic analysis reveals differential roles of viscosity and friction in sickle cell blood

HM Szafraniec, JM Valdez, E Iffrig, WA Lam… - Lab on a Chip, 2022 - pubs.rsc.org
Characterization of blood flow rheology in hematological disorders is critical for
understanding disease pathophysiology. Existing methods to measure blood rheological …

Allosteric control of hemoglobin S fiber formation by oxygen and its relation to the pathophysiology of sickle cell disease

ER Henry, T Cellmer… - Proceedings of the …, 2020 - National Acad Sciences
The pathology of sickle cell disease is caused by polymerization of the abnormal
hemoglobin S upon deoxygenation in the tissues to form fibers in red cells, causing them to …

Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling

DC De Souza, N Hebert, EB Esrick… - Nature …, 2023 - nature.com
We previously reported initial clinical results of post-transcriptional gene silencing of
BCL11A expression (NCT 03282656) reversing the fetal to adult hemoglobin switch. A goal …

The unique magnetic signature of sickle red blood cells: a comparison between the red blood cells of transfused and non-transfused sickle cell disease patients and …

M Weigand, J Gómez-Pastora, J Strayer… - IEEE Transactions …, 2022 - ieeexplore.ieee.org
Sickle cell disease (SCD) is an inherited blood disorder that affects millions of people
worldwide, especially in low-resource regions of the world, where a rapid and affordable test …

[PDF][PDF] Treating sickle cell anemia: a new era dawns

MH Steinberg - American journal of hematology, 2020 - researchgate.net
FIGURE 1 Pathophysiology and HbS polymerization. HbS polymerization (left) initiates the
pathology of disease that has two interrelated limbs. These are hemolytic anemia-a varying …