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Antisense oligonucleotide splicing modulation as a novel Cystic Fibrosis therapeutic approach for the W1282X nonsense mutation.
Oren, Yifat S; Avizur-Barchad, Ofra; Ozeri-Galai, Efrat; Elgrabli, Renana; Schirelman, Meital R; Blinder, Tehilla; Stampfer, Chava D; Ordan, Merav; Laselva, Onofrio; Cohen-Cymberknoh, Malena; Kerem, Eitan; Bear, Christine E; Kerem, Batsheva.
Afiliação
  • Oren YS; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Avizur-Barchad O; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Ozeri-Galai E; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Elgrabli R; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Schirelman MR; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Blinder T; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Stampfer CD; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Ordan M; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel.
  • Laselva O; Department of Medical and Surgical Sciences, University of Foggia, Foggia, Italy; Division of Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON M5G 0A4, Canada.
  • Cohen-Cymberknoh M; Pediatric Pulmonology Unit and CF Center, Hadassah Medical Center and Faculty of Medicine, Hebrew University of Jerusalem, Israel.
  • Kerem E; CF Center, Hadassah Hebrew University Medical Center, Jerusalem, Israel.
  • Bear CE; Division of Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON M5G 0A4, Canada.
  • Kerem B; SpliSense Biohouse Labs, Hadassah Ein Kerem, Jerusalem, Israel; Department of Genetics, The Hebrew University, Jerusalem, Israel. Electronic address: batshevak@savion.huji.ac.il.
J Cyst Fibros ; 21(4): 630-636, 2022 07.
Article em En | MEDLINE | ID: mdl-34972649
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Regulador de Condutância Transmembrana em Fibrose Cística / Fibrose Cística Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Regulador de Condutância Transmembrana em Fibrose Cística / Fibrose Cística Limite: Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article