Context-dependent TGFβ family signalling in cell fate regulation

L Richardson, SG Wilcockson, L Guglielmi… - … Reviews Molecular Cell …, 2023 - nature.com
The transforming growth factor-β (TGFβ) family are a large group of evolutionarily conserved
cytokines whose signalling modulates cell fate decision-making across varying cellular …

[HTML][HTML] Molecular genetics of β-thalassemia: A narrative review

TH Jaing, TY Chang, SH Chen, CW Lin, YC Wen… - Medicine, 2021 - journals.lww.com
Abstract β-thalassemia is a hereditary hematological disease caused by over 350 mutations
in the β-globin gene (HBB). Identifying the genetic variants affecting fetal hemoglobin (HbF) …

Beta thalassemia: new therapeutic options beyond transfusion and iron chelation

I Motta, R Bou-Fakhredin, AT Taher, MD Cappellini - Drugs, 2020 - Springer
Hemoglobinopathies are among the most common monogenic diseases worldwide.
Approximately 1–5% of the global population are carriers for a genetic thalassemia …

[HTML][HTML] Bone morphogenetic protein receptors: Structure, function and targeting by selective small molecule kinase inhibitors

G Sanchez-Duffhues, E Williams, MJ Goumans… - Bone, 2020 - Elsevier
Bone morphogenetic proteins (BMPs) are secreted cytokines that control the fate and
function of many different cell types. They exert their cellular responses via heteromeric …

Myostatin/activin receptor ligands in muscle and the development status of attenuating drugs

BD Rodgers, CW Ward - Endocrine Reviews, 2022 - academic.oup.com
Muscle wasting disease indications are among the most debilitating and often deadly
noncommunicable disease states. As a comorbidity, muscle wasting is associated with …

The use of luspatercept for thalassemia in adults

MD Cappellini, AT Taher - Blood advances, 2021 - ashpublications.org
Luspatercept is an activin receptor ligand trap that has been shown to enhance late-stage
erythropoiesis in animal models of β-thalassemia. A multicenter, international, phase 2 dose …

Development of luspatercept to treat ineffective erythropoiesis

AS Kubasch, P Fenaux, U Platzbecker - Blood advances, 2021 - ashpublications.org
Luspatercept (Reblozyl) was recently approved for treating patients with transfusion-
dependent lower-risk myelodysplastic syndrome (MDS) with ring sideroblasts (RS) and/or …

Genetic retargeting of E3 ligases to enhance CAR T cell therapy

IC Lane, G Kembuan, J Carreiro, MC Kann, W Lin… - Cell Chemical …, 2024 - cell.com
Chimeric antigen receptor (CAR) T cell therapies are medical breakthroughs in cancer
treatment. However, treatment failure is often caused by CAR T cell dysfunction. Additional …

Tumor immune evasion induced by dysregulation of erythroid progenitor cells development

TM Grzywa, M Justyniarska, D Nowis, J Golab - Cancers, 2021 - mdpi.com
Simple Summary Tumor immune evasion is one of the hallmarks of tumor progression that
enables tumor growth despite the activity of the host immune system. It is mediated by …

Activin A: an emerging target for improving cancer treatment?

A Ries, K Schelch, D Falch, L Pany… - Expert Opinion on …, 2020 - Taylor & Francis
Introduction Activin A is involved in the regulation of a surprisingly broad number of
processes that are relevant for cancer development and treatment; it is implicated in cell …