Protein aggregation-inhibition: a therapeutic route from Parkinson's disease to sickle cell anemia

GF Martins, N Galamba - Critical Reviews in Biochemistry and …, 2023 - Taylor & Francis
Protein aggregation is implicated in multiple diseases, so-called proteinopathies, ranging
from neurodegenerative disorders such as Alzheimer's disease and Parkinson's disease …

Recent developments in the use of pyruvate kinase activators as a new approach for treating sickle cell disease

DS Parekh, WA Eaton, SL Thein - Blood, 2024 - ashpublications.org
Pyruvate kinase (PK) is a key enzyme in glycolysis, the sole source of adenosine
triphosphate, which is essential for all energy-dependent activities of red blood cells …

[HTML][HTML] Mocetinostat activates Krüppel-like factor 4 and protects against tissue destruction and inflammation in osteoarthritis

M Kawata, DB McClatchy, JK Diedrich, M Olmer… - JCI insight, 2023 - ncbi.nlm.nih.gov
Osteoarthritis (OA) is the most common joint disorder, and disease-modifying OA drugs
(DMOADs) represent a major need in OA management. Krüppel-like factor 4 (KLF4) is a …

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 …

In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation

AK Leonard, D Furstenau, Z Inam, C Luckett… - Blood …, 2024 - ashpublications.org
Stable, mixed-donor–recipient chimerism after allogeneic hematopoietic stem cell
transplantation (HSCT) for patients with sickle cell disease (SCD) is sufficient for phenotypic …

A framework of computer vision-enhanced microfluidic approach for automated assessment of the transient sickling kinetics in sickle red blood cells

Y Qiang, M Xu, MP Pochron, M Jupelli, M Dao - Frontiers in physics, 2024 - frontiersin.org
The occurrence of vaso-occlusive crisis greatly depends on the competition between the
sickling delay time and the transit time of individual sickle cells, ie, red blood cells from sickle …

Cyclic Peptides as Aggregation Inhibitors for Sickle Cell Disease

V Neto, BL Victor, N Galamba - Journal of Medicinal Chemistry, 2023 - ACS Publications
Sickle cell disease is a missense genetic disorder characterized by the aggregation of deoxy-
HbS into helical fibers that distort erythrocytes into a sickle-like shape. Herein, we …

Label-Free Quantification of DNA Hybridization Kinetics and Affinity Enabled by an Electro-Optical Modulation on Single Nanoparticles

H Zhu, Z Cai, Z Chen, Y Chen, JJ Zhu - Analytical Chemistry, 2023 - ACS Publications
Since the formation of a DNA duplex plays a vital role in gene expression and regulation
across all kinds of organisms, quantifying the interaction during DNA hybridization is …

Functional and multi-omics signatures of mitapivat efficacy upon activation of pyruvate kinase in red blood cells from patients with sickle cell disease

A D'Alessandro, K Le, M Lundt, Q Li, E Dunkelberger… - Blood, 2023 - Elsevier
2, 3-diphosphoglycerate (DPG) in red blood cells (RBCs) stabilizes deoxyhemoglobin to
promote hemoglobin (Hb) oxygen off-loading and counteract hypoxia. In sickle cell disease …

Screen “play” for drug discovery

A Narla, N Mohandas - Proceedings of the National …, 2022 - National Acad Sciences
Sickle cell disease (SCD) is one of the most common and clinically severe inherited
diseases in the world, estimated to affect at least 3 to 6 million people. Greater than 90% of …