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AAV9-mediated SMN gene therapy rescues cardiac desmin but not lamin A/C and elastin dysregulation in Smn2B/- spinal muscular atrophy mice.
Brown, Sharon J; Soltic, Darija; Synowsky, Silvia A; Shirran, Sally L; Chilcott, Ellie; Shorrock, Hannah K; Gillingwater, Thomas H; Yáñez-Muñoz, Rafael J; Schneider, Bernard; Bowerman, Melissa; Fuller, Heidi R.
Affiliation
  • Brown SJ; School of Pharmacy and Bioengineering, Keele University, Keele ST5 5BG, UK.
  • Soltic D; Wolfson Centre for Inherited Neuromuscular Disease, TORCH Building, RJAH Orthopaedic Hospital, Oswestry SY10 7AG, UK.
  • Synowsky SA; School of Pharmacy and Bioengineering, Keele University, Keele ST5 5BG, UK.
  • Shirran SL; Wolfson Centre for Inherited Neuromuscular Disease, TORCH Building, RJAH Orthopaedic Hospital, Oswestry SY10 7AG, UK.
  • Chilcott E; BSRC Mass Spectrometry and Proteomics Facility, University of St Andrews, St Andrews KY16 9ST, UK.
  • Shorrock HK; BSRC Mass Spectrometry and Proteomics Facility, University of St Andrews, St Andrews KY16 9ST, UK.
  • Gillingwater TH; AGCTlab.org, Centre of Gene and Cell Therapy, Centre for Biomedical Sciences, Department of Biological Sciences, School of Life Sciences and the Environment, Royal Holloway University of London, Egham Hill, Egham, Surrey TW20 0EX, UK.
  • Yáñez-Muñoz RJ; Edinburgh Medical School: Biomedical Sciences, Euan MacDonald Centre for Motor Neurone Disease Research, University of Edinburgh, Edinburgh EH8 9XD, UK.
  • Schneider B; Edinburgh Medical School: Biomedical Sciences, Euan MacDonald Centre for Motor Neurone Disease Research, University of Edinburgh, Edinburgh EH8 9XD, UK.
  • Bowerman M; AGCTlab.org, Centre of Gene and Cell Therapy, Centre for Biomedical Sciences, Department of Biological Sciences, School of Life Sciences and the Environment, Royal Holloway University of London, Egham Hill, Egham, Surrey TW20 0EX, UK.
  • Fuller HR; Bertarelli Platform for Gene Therapy, Ecole Polytechnique Fédérale de Lausanne (EPFL), 1202 Geneva, Switzerland.
Hum Mol Genet ; 32(20): 2950-2965, 2023 10 04.
Article in En | MEDLINE | ID: mdl-37498175
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Muscular Atrophy, Spinal / Motor Neurons Limits: Animals / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2023 Type: Article Affiliation country: United kingdom
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Muscular Atrophy, Spinal / Motor Neurons Limits: Animals / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2023 Type: Article Affiliation country: United kingdom