βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells

Lentiviral addition of βT87Q-globin, a modified β-globin with an anti-sickling mutation, is currently being used in gene therapy trials for sickle cell disease (SCD) and β-thalassemia patients. βT87Q-globin interferes with sickle hemoglobin (HbS) polymerization. Here, we generated the SCD mutation i...

Full description

Bibliographic Details
Main Authors: Selami Demirci, Bjorg Gudmundsdottir, Quan Li, Juan J. Haro-Mora, Tina Nassehi, Claire Drysdale, Morgan Yapundich, Jackson Gamer, Fayaz Seifuddin, John F. Tisdale, Naoya Uchida
Format: Article
Language:English
Published: Elsevier 2020-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
sickle cell disease
gene therapy
hemoglobin
immortalized erythrocytes
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120300735

Internet

http://www.sciencedirect.com/science/article/pii/S2329050120300735

Similar Items