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Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
Institution:1. Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA;2. Harvard Program in Biological and Biomedical Sciences, Harvard Medical School, Boston, MA 02115, USA;3. Harvard Program in Virology, Harvard Medical School, Boston, MA 02115, USA;4. Harvard-MIT Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA;5. Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA;6. Harvard Stem Cell Institute, Cambridge, MA 02138, USA;7. Paul F. Glenn Center for the Biology of Aging, Harvard Medical School, Boston, MA 02115, USA;8. Stem Cell Program and Division of Hematology/Oncology, Boston Children’s Hospital, Boston, MA 02115, USA;9. Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children’s Hospital, Harvard Medical School, Boston, MA 02115, USA;10. Department of Organismic and Evolutionary Biology, FAS Center for Systems Biology, Harvard University, Cambridge, MA 02138, USA;11. Section on Islet Cell and Regenerative Biology, Joslin Diabetes Center, Boston, MA 02215, USA;12. Howard Hughes Medical Institute, Chevy Chase, MD 20815, USA
Abstract:
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  • Keywords:directed evolution  AAV capsid engineering  muscle gene therapy  Duchenne muscular dystrophy  X-linked myotubular myopathy  integrin heterodimers  non-human primates  MyoAAV
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