Genzyme Corporation

Avigen, Inc.

Richard T Surosky

Peter C Colosi

Alejandra Elena Arbetman

Michael A Lochrie

Georges Natsoulis

Methods of making and using recombinant AAV virions with decreased immunoreactivity are described. The recombinant AAV virions include mutated capsid proteins or are derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2.

AAV virions with decreased immunoreactivity and uses therefor

A novel targeted nucleotide sequence delivery and integration system is provided. The integration system employs nucleic acid constructs having a targeting sequence capable of binding AAV Rep and a heterologous nucleotide sequence arranged relative to the targeting sequence such that the targeting sequence is capable of directing the integration of the heterologous sequence into a target site in a recipient genome. The system further employs Rep expression products which provide integration functions effective to mediate the site-specific integration of the targeting sequence and the heterologous sequence into the recipient genome. Methods are described, whereby the nucleotide sequence integration system can deliver and efficiently integrate large nucleotide sequences into target sites in recipient cell genomes. Therapeutic methods are also provided, wherein the integration systems are used to insert various therapeutically relevant nucleotide sequences into selected cells from a subject.

Growing community of inventors

Oakland, CA, United States of America

Richard T Surosky