Articles

2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance

Weill Cornell Medical College of Cornell University, New York, USA;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
University of Milan - Fondazione IRCCS Ca' Granda Ospedale Policlinico, Milan, Italy;
Ionis Pharmaceuticals, Carlsbad, CA, USA;
Ionis Pharmaceuticals, Carlsbad, CA, USA;
Ionis Pharmaceuticals, Carlsbad, CA, USA;
Ionis Pharmaceuticals, Carlsbad, CA, USA;
Ionis Pharmaceuticals, Carlsbad, CA, USA;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
Dept. of Molecular Genetics Thalassaemia, Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;
University of Milan - Fondazione IRCCS Ca' Granda Ospedale Policlinico, Milan, Italy;
Graduate School of Medical Sciences, Weill Cornell Medical College of Cornell University, New York;
Dept. of Molecular Genetics Thalassaemia, Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;
Dept. of Molecular Genetics Thalassaemia, Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
The Children Hospital at Montefiore, Bronx, NY, USA
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
University of Milan - Fondazione IRCCS Ca' Granda Ospedale Policlinico, Milan, Italy;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA;
Haematologica Early view May 21, 2020 https://doi.org/10.3324/haematol.2019.226852