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

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.

Afiliação

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 em En | MEDLINE | ID: mdl-37498175

Assuntos

Neurônios Motores; Atrofia Muscular Espinal; Humanos; Camundongos; Animais; Neurônios Motores/metabolismo; Desmina/genética; Desmina/metabolismo; Elastina/genética; Lamina Tipo A/genética; Lamina Tipo A/metabolismo; Atrofia Muscular Espinal/genética; Atrofia Muscular Espinal/terapia; Atrofia Muscular Espinal/patologia; Terapia Genética; Modelos Animais de Doenças; Proteína 1 de Sobrevivência do Neurônio Motor/genética; Proteína 1 de Sobrevivência do Neurônio Motor/metabolismo

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Atrofia Muscular Espinal / Neurônios Motores Limite: Animals / Humans Idioma: En Revista: Hum Mol Genet Assunto da revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Reino Unido

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