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S06-098: Novel AAV capsids with new transduction and nonimmune properties
Stanford University
Gene therapy
Genetic disorders
Target Identification or Validation
Researchers from Dr. Mark Kay's laboratory at Stanford University have merged desirable qualities of multiple natural AAV isolates by an adapted DNA family shuffling technology to create a complex library of hybrid capsids from eight different wild-type viruses. One of the capsids was vectorized and used to express the human transgene factor IX in mice. This AAV vector was found to be as robust or better than the best AAV vector identified in nature to date. Moreover, this capsid does not react with the human immune response. Finally, this capsid is useful for transducing cells in culture, which has been problematic for many of the AAV vectors studied to date.
US 8574583