Gene therapy for spinal muscular atrophy: perspectives on the possibility of optimizing SMN1 delivery to correct all neurological and systemic perturbations.

Brown, Sharon J; Yáñez-Muñoz, Rafael J; Fuller, Heidi R

Brown, Sharon J; Yáñez-Muñoz, Rafael J; Fuller, Heidi R.

Afiliação

Brown SJ; School of Pharmacy and Bioengineering, Keele University, Keele, UK (Brown SJ, Fuller HR).
Yáñez-Muñoz RJ; Wolfson Center for Inherited Neuromuscular Disease, TORCH Building, RJAH Orthopaedic Hospital, Oswestry, UK (Brown SJ, Fuller HR).
Fuller HR; AGCTlab.org, Center of Gene and Cell Therapy, Department of Biological Sciences, School of Life Sciences and the Environment, Royal Holloway University of London, Egham, UK (Yáñez-Muñoz RJ).

Neural Regen Res ; 20(7): 2011-2012, 2025 Jul 01.

Article em En | MEDLINE | ID: mdl-39254562

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Neural Regen Res Ano de publicação: 2025 Tipo de documento: Article

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Neural Regen Res Ano de publicação: 2025 Tipo de documento: Article