Next-generation muscle-directed gene therapy by in silico vector design
Author:
Publisher
Springer Science and Business Media LLC
Subject
General Physics and Astronomy,General Biochemistry, Genetics and Molecular Biology,General Chemistry
Link
http://www.nature.com/articles/s41467-018-08283-7.pdf
Reference67 articles.
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3. Mendell, J. R. et al. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713–1722 (2017).
4. Seymour, L. W. & Thrasher, A. J. Gene therapy matures in the clinic. Nat. Biotechnol. 30, 588–593 (2012).
5. Verma, I. M. Gene therapy that works. Science 341, 853–855 (2013).
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