2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance
Alisa Dong(Children's Hospital of Philadelphia), Laura Breda(Children's Hospital of Philadelphia), Yasuhiro Ikawa(Children's Hospital of Philadelphia), Carla Casu(Children's Hospital of Philadelphia), Shuling Guo(Ionis Pharmaceuticals (United States)), Marina Kleanthous(Cyprus Institute of Neurology and Genetics), Danuta Jarocha(Jagiellonian University), Paola Delbini(University of Milan), Stefano Rivella(Children's Hospital of Philadelphia), Valentina Ghiaccio(Children's Hospital of Philadelphia), Raechel Peralta(Ionis Pharmaceuticals (United States)), Soteroula Christou(Archbishop Makarios III Hospital), Osheiza Abdulmalik(Children's Hospital of Philadelphia), Maria Domenica Cappellini(Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico), Maxwell Chappell(Children's Hospital of Philadelphia), Andy Watt(Ionis Pharmaceuticals (United States)), Sagar Damle(Ionis Pharmaceuticals (United States)), Kim Smith‐Whitley(Children's Hospital of Philadelphia), Irene Motta(Médecins Sans Frontières), Connie Chen(Cornell University), Deepa Manwani(Albert Einstein College of Medicine), Susan M. Freier(Ionis Pharmaceuticals (United States)), Coralea Stephanou(Agruicultural Research Institute)
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