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A Novel Triple-Mutant AAV6 Capsid Induces Rapid and Potent Transgene Expression in the Muscle and Respiratory Tract of Mice
Gene therapy for the treatment of genetic disorders has demonstrated considerable therapeutic success in clinical trials. Among the most effective and commonly used gene delivery vectors are those based on adeno-associated virus (AAV). Despite these advances in clinical gene therapy, further improve...
Autores principales: | van Lieshout, Laura P., Domm, Jakob M., Rindler, Tara N., Frost, Kathy L., Sorensen, Debra L., Medina, Sarah J., Booth, Stephanie A., Bridges, James P., Wootton, Sarah K. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6054702/ https://www.ncbi.nlm.nih.gov/pubmed/30038936 http://dx.doi.org/10.1016/j.omtm.2018.04.005 |
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