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.

主权项

1. An improved adeno-associated virus (AAV) vector comprising a VP1 capsid protein comprising one or more lysine substitutions, said vector further comprising a minigene comprising AAV inverted terminal repeats and a heterologous nucleic acid sequence operably linked to regulatory sequences which direct expression of a product from the heterologous nucleic acid sequence in a host cell, said lysine substitution being effective to inhibit ubiquitination of said capsid protein, thereby increasing transduction of said AAV vector into a target cell.