Low levels of CFTR gene transfer with F/HN pseudotyped lentivirus are sufficient to generate ion transport correction in airway cell cultures from cystic fibrosis patients

Autor: Sergijenko, A, Moiseenko, A, Pineault, K, Nafchi, NAM, Chan, M, Gamlen, T, Gill, D, Hyde, S, Kreuz, S, Griesenbach, U, Alton, EWFW

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______1064::eed21b000f172b9b8940d13de98ba29c
https://ora.ox.ac.uk/objects/uuid:ffcd8d4e-ed93-4c63-b80a-4345e88bfe0c

Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo.

Autor: Nafchi NAM; 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, TW20 0EX, UK., Chilcott EM; 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, TW20 0EX, UK., Brown S; School of Pharmacy and Bioengineering, Keele University, Staffordshire, ST5 5BG, UK.; Wolfson Centre for Inherited Neuromuscular Disease, TORCH Building, RJAH Orthopaedic Hospital, Oswestry, SY10 7AG, UK., Fuller HR; School of Pharmacy and Bioengineering, Keele University, Staffordshire, ST5 5BG, UK.; Wolfson Centre for Inherited Neuromuscular Disease, TORCH Building, RJAH Orthopaedic Hospital, Oswestry, SY10 7AG, UK., Bowerman M; Wolfson Centre for Inherited Neuromuscular Disease, TORCH Building, RJAH Orthopaedic Hospital, Oswestry, SY10 7AG, UK.; School of Medicine, Keele University, Staffordshire, ST5 5BG, UK., Yáñez-Muñoz RJ; 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, TW20 0EX, UK. rafael.yanez@royalholloway.ac.uk.

Publikováno v: Gene therapy [Gene Ther] 2023 Dec; Vol. 30 (12), pp. 812-825. Date of Electronic Publication: 2023 Jun 15.