Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain

@article{Sullivan2018RationallyDA,
  title={Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain},
  author={Jennifer L. Sullivan and Lisa M. Stanek and Michael J. Lukason and Jie Bu and Shayla R. Osmond and Elizabeth A. Barry and Catherine R. O’Riordan and Lamya S. Shihabuddin and Seng H. Cheng and Abraham Scaria},
  journal={Gene Therapy},
  year={2018},
  volume={25},
  pages={205-219}
}
The successful application of adeno-associated virus (AAV) gene delivery vectors as a therapeutic paradigm will require efficient gene delivery to the appropriate cells in affected organs. In this study, we utilized a rational design approach to introduce modifications to the AAV2 and AAVrh8R capsids and the resulting variants were evaluated for transduction activity in the retina and brain. The modifications disrupted either capsid/receptor binding or altered capsid surface charge… CONTINUE READING