2'-O-Methoxyethyl Splice-Switching Oligos to Reverse Splicing from IVS2-745 β-Thalassemia Patient Cells: A Foundation for Potential Therapies
Valentina Ghiaccio(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), Marina Kleanthous(Cyprus Institute of Neurology and Genetics), Paola Delbini(University of Milan), Stefano Rivella(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), Alisa Dong(Children's Hospital of Philadelphia), Maxwell Chappell(Children's Hospital of Philadelphia), Andy Watt(Ionis Pharmaceuticals (United States)), Kim Smith‐Whitley(Children's Hospital of Philadelphia), Irene Motta(Médecins Sans Frontières), Connie Chen(Cornell University), Shuling Guo(University of California, Los Angeles), Deepa Manwani(Albert Einstein College of Medicine), Susan M. Freier(Ionis Pharmaceuticals (United States)), Coralea Stephanou(Agruicultural Research Institute)
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