βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells
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
Abstract
-globin expression, resulting in an anti-sickling effect. Our findings should be helpful to understand the anti-sickling effects of therapeutic genes in SCD gene therapy.
Topics & Concepts
Ex vivoHemoglobinGlobinIn vivoGenetic enhancementHemoglobinopathyFetal hemoglobinGeneBiologyHemolytic anemiaImmunologyGeneticsBiochemistryPregnancyFetusHemoglobinopathies and Related DisordersIron Metabolism and DisordersErythrocyte Function and Pathophysiology