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...

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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Demirci, Selami, Gudmundsdottir, Bjorg, Li, Quan, Haro-Mora, Juan J., Nassehi, Tina, Drysdale, Claire, Yapundich, Morgan, Gamer, Jackson, Seifuddin, Fayaz, Tisdale, John F., Uchida, Naoya
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7210457/
https://ncbi.nlm.nih.gov/pubmed/32405513
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.04.013
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