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The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy.
Scaglioni, Dominic; Catapano, Francesco; Ellis, Matthew; Torelli, Silvia; Chambers, Darren; Feng, Lucy; Beck, Matthew; Sewry, Caroline; Monforte, Mauro; Harriman, Shawn; Koenig, Erica; Malhotra, Jyoti; Popplewell, Linda; Guglieri, Michela; Straub, Volker; Mercuri, Eugenio; Servais, Laurent; Phadke, Rahul; Morgan, Jennifer; Muntoni, Francesco.
Afiliação
  • Scaglioni D; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford St, London, WC1N 1EH, UK.
  • Catapano F; NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
  • Ellis M; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford St, London, WC1N 1EH, UK.
  • Torelli S; NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
  • Chambers D; Dubowitz Neuromuscular Centre, UCL Queen Square Institute of Neurology and Great Ormond Street, London, UK.
  • Feng L; Department of Neurodegenerative Diseases, UCL Queen Square Institute of Neurology, London, UK.
  • Beck M; School of Cancer Sciences, University of Southampton, Southampton, UK.
  • Sewry C; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford St, London, WC1N 1EH, UK.
  • Monforte M; NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
  • Harriman S; Dubowitz Neuromuscular Centre, UCL Queen Square Institute of Neurology and Great Ormond Street, London, UK.
  • Koenig E; Dubowitz Neuromuscular Centre, UCL Queen Square Institute of Neurology and Great Ormond Street, London, UK.
  • Malhotra J; Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, 30 Guilford St, London, WC1N 1EH, UK.
  • Popplewell L; NIHR Great Ormond Street Hospital Biomedical Research Centre, UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
  • Guglieri M; Dubowitz Neuromuscular Centre, UCL Queen Square Institute of Neurology and Great Ormond Street, London, UK.
  • Straub V; RJAH Orthopaedic Hospital NHS Trust, Oswestry, UK.
  • Mercuri E; Paediatric Neurology and Centro Clinico Nemo, Catholic University and Policlinico Gemelli, Fondazione Policlinico Universitario Agostino Gemelli IRCSS, Rome, Italy.
  • Servais L; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Phadke R; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Morgan J; Sarepta Therapeutics, Inc., Cambridge, MA, USA.
  • Muntoni F; Centre of Gene and Cell Therapy and Centre for Biomedical Sciences, Royal Holloway, University of London, Egham, UK.
Acta Neuropathol Commun ; 9(1): 7, 2021 01 06.
Article em En | MEDLINE | ID: mdl-33407808
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Regeneração / Distrofina / Oligonucleotídeos Antissenso / Músculo Esquelético / Distrofia Muscular de Duchenne Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Oligonucleotídeos / Regeneração / Distrofina / Oligonucleotídeos Antissenso / Músculo Esquelético / Distrofia Muscular de Duchenne Idioma: En Ano de publicação: 2021 Tipo de documento: Article