Adult Hemoglobin Production, Chain Rebalance, and Splice Correction in IVS2-745 Beta-Thalassemia Patient Cells Using 2'-O-Methoxyethyl Splice-Switching Oligos
Alisa Dong(Children's Hospital of Philadelphia), Stefano Rivella(Children's Hospital of Philadelphia), Marina Kleanthous(Cyprus Institute of Neurology and Genetics), Paola Delbini(University of Milan), 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), Laura Breda(Children's Hospital of Philadelphia), Irene Motta(Médecins Sans Frontières), Coralea Stephanou(Agruicultural Research Institute), Connie Chen(Cornell University), Shuling Guo(Ionis Pharmaceuticals (United States))
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