Composition and methods for highly efficient gene transfer using aav capsid variants

摘要

Improved compositions and methods for AAV mediated gene therapy are disclosed. In particular, the present invention relates to an AAV vector comprising a VP1 capsid protein comprising one or more amino-acid substitutions and thereby providing improved characteristics compared to an AAV vector comprising the VP1 capsid protein without said substitutions. The AAV vectors described herein exhibit increased transduction efficiency when compared to known AAV serotypes.