caa a22 4500 397573421 CHVBK 20180308164848.0 cr unu---uuuuu 161202e199609 xx s 000 0 eng 10.1093/hmg/5.Supplement_1.1397 doi (NATIONALLICENCE)oxford-10.1093/hmg/5.Supplement_1.1397 Applications of gene therapy to the CNS [Elektronische Daten] [Ulrike Blömer, Luigi Naldini, Inder M. Verma, Didier Trono, Fred H. Gage] Gene therapy is a new method with potential for treating a broad range of acquired and inherited neurologic diseases, where the causative gene defect or deletion has been identified. In addition to gene replacement the application of gene products that reduce cellular dysfunction or death represent new therapeutic options. Gene transfer techniques to express novel proteins using different viral vectors in vitro and in vivo, as well as animal models and human trials will be reviewed in this article. We will focus on a new lentiviral vector as a recent gene transfer method and degenerative disorders of the CNS, and their related model systems. © 1996 Oxford University Press Blömer Ulrike The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut Naldini Luigi The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut Verma Inder M. The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut Trono Didier The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut Gage Fred H. The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut Human Molecular Genetics Oxford University Press 5/Supplement_1(1996-09), 1397-1404 0964-6906 5:Supplement_1<1397 1996 5 hmg https://doi.org/10.1093/hmg/5.Supplement_1.1397 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1093/hmg/5.Supplement_1.1397 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Blömer Ulrike The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut NATIONALLICENCE 700 1- Naldini Luigi The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut NATIONALLICENCE 700 1- Verma Inder M. The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut NATIONALLICENCE 700 1- Trono Didier The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut NATIONALLICENCE 700 1- Gage Fred H. The Salk Institute for Biological Studies, 10010 North Torrey Pines Rd., La Jolla, CA, 92037-1099, USA aut NATIONALLICENCE 773 0- Human Molecular Genetics Oxford University Press 5/Supplement_1(1996-09), 1397-1404 0964-6906 5:Supplement_1<1397 1996 5 hmg Metadata rights reserved CC BY-NC-4.0 http://creativecommons.org/licenses/by-nc/4.0 nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-oxford