A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence
Recombinant adeno-associated virus (rAAV) is currently the best vector for gene delivery into the skeletal muscle. However, the 5-kb packaging size of this virus is a major obstacle for large gene transfer. This past decade, many different strategies were developed to circumvent this issue (concatem...
Main Authors: | Marina Pryadkina, William Lostal, Nathalie Bourg, Karine Charton, Carinne Roudaut, Matthew L Hirsch, Isabelle Richard |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2015-01-01
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Series: | Molecular Therapy: Methods & Clinical Development |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050116300213 |
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