Therapeutic strategies for sickle cell disease: towards a multi-agent approach

MJ Telen, P Malik, GM Vercellotti - Nature reviews Drug discovery, 2019 - nature.com
For over 100 years, clinicians and scientists have been unravelling the consequences of the
A to T substitution in the β-globin gene that produces haemoglobin S, which leads to the …

LSD1: biologic roles and therapeutic targeting

A Maiques-Diaz, TCP Somervaille - Epigenomics, 2016 - Taylor & Francis
LSD1 (KDM1A; BHC110; AOF2) was the first protein reported to exhibit histone demethylase
activity and has since been shown to have multiple essential roles in mammalian biology …

An extra-erythrocyte role of haemoglobin body in chondrocyte hypoxia adaption

F Zhang, B Zhang, Y Wang, R Jiang, J Liu, Y Wei… - Nature, 2023 - nature.com
Although haemoglobin is a known carrier of oxygen in erythrocytes that functions to transport
oxygen over a long range, its physiological roles outside erythrocytes are largely elusive …

Tranylcypromine based lysine-specific demethylase 1 inhibitor: summary and perspective

XJ Dai, Y Liu, XP **ong, LP Xue… - Journal of medicinal …, 2020 - ACS Publications
Histone lysine-specific demethylase 1 (LSD1/KDM1A) has become an important and
promising anticancer target since it was first identified in 2004 and specially demethylates …

Thalassemias: from gene to therapy

G De Simone, A Quattrocchi, B Mancini… - Molecular Aspects of …, 2022 - Elsevier
Abstract Thalassemias (α, β, γ, δ, δβ, and εγδβ) are the most common genetic disorders
worldwide and constitute a heterogeneous group of hereditary diseases characterized by …

Emerging disease-modifying therapies for sickle cell disease

MA Carden, J Little - Haematologica, 2019 - pmc.ncbi.nlm.nih.gov
Sickle cell disease afflicts millions of people worldwide and approximately 100,000
Americans. Complications are myriad and arise as a result of complex pathological …

Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies

DS Vinjamur, DE Bauer… - British journal of …, 2018 - Wiley Online Library
The major β‐haemoglobinopathies, sickle cell disease and β‐thalassaemia, represent the
most common monogenic disorders worldwide and a steadily increasing global disease …

PGC-1α agonism induces fetal hemoglobin and exerts antisickling effects in sickle cell disease

Y Sun, H Benmhammed, S Al Abdullatif, A Habara… - Science …, 2024 - science.org
Sickle cell disease is a growing health burden afflicting millions around the world. Clinical
observation and laboratory studies have shown that the severity of sickle cell disease is …

Fetal haemoglobin in sickle-cell disease: from genetic epidemiology to new therapeutic strategies

G Lettre, DE Bauer - The Lancet, 2016 - thelancet.com
Sickle-cell disease affects millions of individuals worldwide, but the global incidence is
concentrated in Africa. The burden of sickle-cell disease is expected to continue to rise over …

A specific G9a inhibitor unveils BGLT3 lncRNA as a universal mediator of chemically induced fetal globin gene expression

S Takase, T Hiroyama, F Shirai, Y Maemoto… - Nature …, 2023 - nature.com
Sickle cell disease (SCD) is a heritable disorder caused by β-globin gene mutations.
Induction of fetal γ-globin is an established therapeutic strategy. Recently, epigenetic …