Epigenetic inactivation of ERF reactivates γ-globin expression in β-thalassemia.
Sign Up to like & getrecommendations! Published in 2021 at "American journal of human genetics"
DOI: 10.1016/j.ajhg.2021.03.005
Abstract: The fetal-to-adult hemoglobin switch is regulated in a developmental stage-specific manner and reactivation of fetal hemoglobin (HbF) has therapeutic implications for treatment of β-thalassemia and sickle cell anemia, two major global health problems. Although significant… read more here.
Keywords: hbf; erf; epigenetic inactivation; thalassemia ... See more keywords
Sustained enhancement of OCTN1 transporter expression in association with hydroxyurea induced γ-globin expression in erythroid progenitors.
Sign Up to like & getrecommendations! Published in 2017 at "Experimental hematology"
DOI: 10.1016/j.exphem.2016.09.001
Abstract: The clinical benefits of hydroxyurea (HU) treatment in patients with sickle cell disease (SCD) are due largely to increased γ-globin expression. However, mechanisms that control γ-globin expression by HU in erythroid progenitors are incompletely understood. Here,… read more here.
Keywords: octn1; expression erythroid; expression; globin expression ... See more keywords
Reactivation of γ-globin expression using a minicircle DNA system to treat β-thalassemia.
Sign Up to like & getrecommendations! Published in 2022 at "Gene"
DOI: 10.1016/j.gene.2022.146289
Abstract: Reactivation of fetal hemoglobin by editing the B-cell lymphoma/leukemia 11A (BCL11A) erythroid enhancer is an effective gene therapy for β-thalassemia. Using the CRISPR/Cas9 system, fetal γ-globin expression can be robustly reactivated to mitigate the clinical… read more here.
Keywords: dna; globin expression; expression; reactivation globin ... See more keywords
Restoration of β-globin expression with optimally designed lentiviral vector for β-thalassemia treatment in Chinese patients.
Sign Up to like & getrecommendations! Published in 2020 at "Human gene therapy"
DOI: 10.1089/hum.2020.204
Abstract: β-thalassemia is one of the most prevalent genetic diseases worldwide. The current treatment for β-thalassemia is allogeneic hematopoietic stem cell transplantation (HSCT), which is limited due to lack of matched donors. Gene therapy has been… read more here.
Keywords: treatment; thalassemia; optimally designed; gene ... See more keywords
Oridonin enhances γ‑globin expression in erythroid precursors from patients with β‑thalassemia via activation of p38 MAPK signaling.
Sign Up to like & getrecommendations! Published in 2019 at "Molecular medicine reports"
DOI: 10.3892/mmr.2019.10848
Abstract: Upregulation of fetal hemoglobin expression can alleviate the severity of β‑thalassaemia. This study aimed to investigate the effects of Oridonin (ORI, a diterpenoid compound) on γ‑globin expression in human erythroid precursor cells and the potential… read more here.
Keywords: p38 mapk; globin expression; expression; erythroid precursor ... See more keywords