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A framework for individualized splice-switching oligonucleotide therapy.
Kim, Jinkuk; Woo, Sijae; de Gusmao, Claudio M; Zhao, Boxun; Chin, Diana H; DiDonato, Renata L; Nguyen, Minh A; Nakayama, Tojo; Hu, Chunguang April; Soucy, Aubrie; Kuniholm, Ashley; Thornton, Jennifer Karlin; Riccardi, Olivia; Friedman, Danielle A; El Achkar, Christelle Moufawad; Dash, Zane; Cornelissen, Laura; Donado, Carolina; Faour, Kamli N W; Bush, Lynn W; Suslovitch, Victoria; Lentucci, Claudia; Park, Peter J; Lee, Eunjung Alice; Patterson, Al; Philippakis, Anthony A; Margus, Brad; Berde, Charles B; Yu, Timothy W.
Afiliação
  • Kim J; Graduate School of Medical Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea. jinkuk@kaist.ac.kr.
  • Woo S; Biomedical Research Center, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea. jinkuk@kaist.ac.kr.
  • de Gusmao CM; KI for Health Science and Technology, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea. jinkuk@kaist.ac.kr.
  • Zhao B; Center for Epidemic Preparedness, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea. jinkuk@kaist.ac.kr.
  • Chin DH; Graduate School of Medical Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea.
  • DiDonato RL; Department of Neurology, Boston Children's Hospital, Boston, MA, USA.
  • Nguyen MA; Postgraduate School of Medical Science, University of Campinas (UNICAMP), São Paulo, Brazil.
  • Nakayama T; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Hu CA; Manton Center for Orphan Disease Research, Boston Children's Hospital, Boston, MA, USA.
  • Soucy A; Department of Pediatrics, Boston Children's Hospital, Boston, MA, USA.
  • Kuniholm A; Program in Medical and Population Genetics, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
  • Thornton JK; Harvard Medical School, Boston, MA, USA.
  • Riccardi O; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Friedman DA; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • El Achkar CM; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Dash Z; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Cornelissen L; Harvard Medical School, Boston, MA, USA.
  • Donado C; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Faour KNW; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Bush LW; Institutional Center for Clinical and Translational Research, Boston Children's Hospital, Boston, MA, USA.
  • Suslovitch V; Ataxia Telangiectasia Children's Project, Coconut Creek, FL, USA.
  • Lentucci C; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Park PJ; Department of Neurology, Boston Children's Hospital, Boston, MA, USA.
  • Lee EA; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Patterson A; Department of Neurology, Boston Children's Hospital, Boston, MA, USA.
  • Philippakis AA; Harvard Medical School, Boston, MA, USA.
  • Margus B; Division of Genetics and Genomics, Boston Children's Hospital, Boston, MA, USA.
  • Berde CB; Department of Anesthesiology, Critical Care and Pain Medicine, Boston Children's Hospital, Boston, MA, USA.
  • Yu TW; Department of Anesthesiology, Critical Care and Pain Medicine, Boston Children's Hospital, Boston, MA, USA.
Nature ; 619(7971): 828-836, 2023 Jul.
Article em En | MEDLINE | ID: mdl-37438524
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Ataxia Telangiectasia / Splicing de RNA Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Revista: Nature Ano de publicação: 2023 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: Ataxia Telangiectasia / Splicing de RNA Tipo de estudo: Observational_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Revista: Nature Ano de publicação: 2023 Tipo de documento: Article