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2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 ß-thalassemia patient cells restoring HbA production and chain rebalance.
Dong, Alisa; Ghiaccio, Valentina; Motta, Irene; Guo, Shuling; Peralta, Raechel; Freier, Susan M; Watt, Andy; Damle, Sagar; Ikawa, Yasuhiro; Jarocha, Danuta; Chappell, Maxwell; Stephanou, Coralea; Delbini, Paola; Chen, Connie; Christou, Soteroula; Kleanthous, Marina; Smith-Whitley, Kim; Manwani, Deepa; Casu, Carla; Abdulmalik, Osheiza; Cappellini, Maria Domenica; Rivella, Stefano; Breda, Laura.
Afiliação
  • Dong A; Weill Cornell Medical College of Cornell University, New York, USA.
  • Ghiaccio V; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Motta I; University of Milan - Fondazione IRCCS Ca' Granda Ospedale Policlinico, Milan, Italy.
  • Guo S; Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Peralta R; Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Freier SM; Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Watt A; Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Damle S; Ionis Pharmaceuticals, Carlsbad, CA, USA.
  • Ikawa Y; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Jarocha D; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Chappell M; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Stephanou C; Dept. of Molecular Genetics Thalassaemia, Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Delbini P; University of Milan - Fondazione IRCCS Ca' Granda Ospedale Policlinico, Milan, Italy.
  • Chen C; Graduate School of Medical Sciences, Weill Cornell Medical College of Cornell University, New York.
  • Christou S; Dept. of Molecular Genetics Thalassaemia, Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Kleanthous M; Dept. of Molecular Genetics Thalassaemia, Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus.
  • Smith-Whitley K; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Manwani D; The Children Hospital at Montefiore, Bronx, NY, USA.
  • Casu C; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Abdulmalik O; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Cappellini MD; University of Milan - Fondazione IRCCS Ca' Granda Ospedale Policlinico, Milan, Italy.
  • Rivella S; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
  • Breda L; Department of Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, USA.
Haematologica ; 106(5): 1433-1442, 2019 May 01.
Article em En | MEDLINE | ID: mdl-32439726
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Haematologica Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Haematologica Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Estados Unidos