COMPOSITION AND METHODS FOR HIGHLY EFFICIENT GENE TRANSFER USING AAV CAPSID VARIANTS

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专利权人:

发明人:

申请号：

EP13778449.2

公开号：

EP2839014A1

申请日:

2013.04.18

申请国别(地区):

EP

年份:

2015

代理人:

摘要：

Compositions and methods for AAV mediated gene therapy are disclosed. AAV vectors comprise protein capsid variants comprising therapeutically beneficial transgenes. AAV variants are provided which exhibit increased transduction efficiency when compared to AAV serotypes (e.g., AAV1, AAV2, AAV8, AAV-rh74), which lack the modifications disclosed herein. Such improved vectors are useful for transduction of a variety of tissues.

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