Viral Vectors in Gene Replacement Therapy
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Published:2023-12
Issue:12-13
Volume:88
Page:2157-2178
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ISSN:0006-2979
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Container-title:Biochemistry (Moscow)
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language:en
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Short-container-title:Biochemistry Moscow
Author:
Minskaia Ekaterina,Galieva Alima,Egorov Alexander D.,Ivanov Roman,Karabelsky Alexander
Publisher
Pleiades Publishing Ltd
Subject
Biochemistry,General Medicine,Biochemistry, Genetics and Molecular Biology (miscellaneous),Biophysics,Geriatrics and Gerontology
Reference197 articles.
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https://doi.org/10.1136/ard.2008.089375. 5. Russell, S., Bennett, J., Wellman, J. A., Chung, D. C., Yu, Z. F., Tillman, A., Wittes, J., Pappas, J., Elci, O., McCague, S., et al. (2017) Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial, Lancet, 390, 849-860,
https://doi.org/10.1016/S0140-6736(17)31868-8.
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