Humanized Viral Vectors and Methods of Use Thereof
First Claim
1. A humanized viral vector encoding a therapeutic molecule of interest, said vector comprising at least one human surface molecule thereby reducing antibody binding and clearance via immune surveillance.
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Abstract
The present invention provides humanized viral vectors and methods of use thereof for delivery of transgenes or therapeutic nucleic acids to human subjects. Humanized viral vectors are modified from known viral vectors such as those based on AAV by coating their surface with a human protein such as human serum albumin and optionally a lipid coating or formulation, so that the foreign or non-human nature of the vector is masked. The coating is performed in a manner that reduces or prevents binding of antibodies to the vector surface, thereby reducing or preventing antibody-mediated clearance of vector, but still allowing the vector to transduce target cells and achieve therapeutic gene transfer. Such humanized vectors therefore evade pre-existing immune surveillance, reduce immune responses, and achieve therapeutic gene transfer in the presence of pre-existing antibodies to the viral vector.
32 Citations
21 Claims
- 1. A humanized viral vector encoding a therapeutic molecule of interest, said vector comprising at least one human surface molecule thereby reducing antibody binding and clearance via immune surveillance.
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21. A pharmaceutical preparation comprising a plurality of viral vectors encapsulated in a lipid formulation studded with human surface proteins and optionally at least one carbohydrate moiety.
Specification