Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia

by Tiwaporn Nualkaew, Karine Sii-Felice, Marie Giorgi, Bradley McColl, Julie Gouzil, Astrid Glaser, Hsiao P.J. Voon, Hsin Y. Tee, George Grigoriadis, Saovaros Svasti, Suthat Fucharoen, Suradej Hongeng, Philippe Leboulch, Emmanuel Payen, Jim Vadolas
Published 2022

ATR-X syndrome protein targets tandem repeats and influences allele-specific expression in a size-dependent manner

by Martin J. Law, Karen M. Lower, Hsiao P.J. Voon, Jim R. Hughes, David Garrick, Vip Viprakasit, Matthew Mitson, Marco De Gobbi, Marco Marra, Andrew Morris, Aaron Abbott, Steven P. Wilder, Stephen Taylor, Guilherme M. Santos, Joe Cross, Helena Ayyub, Steven Jones, Jiannis Ragoussis, Daniela Rhodes, Ian Dunham, Douglas R. Higgs, Richard J. Gibbons
Published 2018