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Exon-Skipping Oligonucleotides Restore Functional Collagen VI by Correcting a Common COL6A1 Mutation in Ullrich CMD.
Aguti, Sara; Bolduc, Véronique; Ala, Pierpaolo; Turmaine, Mark; Bönnemann, Carsten G; Muntoni, Francesco; Zhou, Haiyan.
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  • Aguti S; The Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London WC1N 1EH, UK.
  • Bolduc V; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland MD 20892, USA.
  • Ala P; The Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London WC1N 1EH, UK.
  • Turmaine M; Division of Biosciences, University College London, Gower Street, London WC1E 6BT, UK.
  • Bönnemann CG; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland MD 20892, USA.
  • Muntoni F; The Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, 30 Guilford Street, London WC1N 1EH, UK; NIHR Great Ormond Street Hospital Biomedical Research C
  • Zhou H; NIHR Great Ormond Street Hospital Biomedical Research Centre, London WC1N 1EH, UK; Genetics and Genomic Medicine Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK. Electronic address: haiyan.zhou@ucl.ac.uk.
Mol Ther Nucleic Acids ; 21: 205-216, 2020 Sep 04.
Article en En | MEDLINE | ID: mdl-32585628
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Texto completo: 1 Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: Mol Ther Nucleic Acids Año: 2020 Tipo del documento: Article País de afiliación: Reino Unido
Texto completo
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Texto completo: 1 Banco de datos: MEDLINE Tipo de estudio: Prognostic_studies Idioma: En Revista: Mol Ther Nucleic Acids Año: 2020 Tipo del documento: Article País de afiliación: Reino Unido