Author:
Minskaia Ekaterina,Galieva Alima,Egorov Alexander D.,Ivanov Roman,Karabelsky Alexander
Subject
Biochemistry,General Medicine,Biochemistry, Genetics and Molecular Biology (miscellaneous),Biophysics,Geriatrics and Gerontology
Reference197 articles.
1. Mahoney, A. L. G., Nassif, N. T., O’Brien, B. A., and Simpson, A. M. (2022) Viral Vectors in Gene Therapy and Clinical Applications in Molecular Cloning, IntechOpen.
2. Flotte, T., Carter, B., Conrad, C., Guggino, W., Reynolds, T., Rosenstein, B., Taylor, G., Walden, S., and Wetzel, R. (1996) A phase I study of an adeno-associated Virus-CFTR gene vector in adult CF patients with mild lung disease, Hum. Gene Ther., 7, 1145-1159,
https://doi.org/10.1089/hum.1996.7.9-1145.
3. Manno, C. S., Arruda, V. R., Pierce, G. F., Glader, B., Ragni, M., Rasko, J., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response, Nat. Med., 12, 342-347,
https://doi.org/10.1038/nm1358.
4. Mease, P. J., Hobbs, K., Chalmers, A., El-Gabalawy, H., Bookman, A., Keystone, E., Furst, D. E., Anklesaria, P., and Heald, A. E. (2009) Local delivery of a recombinant adenoassociated vector containing a tumour necrosis factor α antagonist gene in inflammatory arthritis: A phase 1 dose-escalation safety and tolerability study, Ann. Rheum. Dis., 68, 1247-1254,
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.