Biodistribution and Safety Profile of Recombinant Adeno-Associated Virus Serotype 6 Vectors following Intravenous Delivery

Autor Stone, Daniel, Liu, Ying, Li, Zong-Yi, Strauss, Robert, Finn, Eric E., Allen, James M., Chamberlain, Jeffrey S., Lieber, André
Vydáno 2008

Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

Autor Wang, Zejing, Storb, Rainer, Halbert, Christine L, Banks, Glen B., Butts, Tiffany M., Finn, Eric E., Allen, James M., Miller, A. Dusty, Chamberlain, Jeffrey S., Tapscott, Stephen J.
Vydáno 2012

Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy

Autor Ho, Peggy P., Lahey, Lauren J., Mourkioti, Foteini, Kraft, Peggy E., Filareto, Antonio, Brandt, Moritz, Magnusson, Klas E. G., Finn, Eric E., Chamberlain, Jeffrey S., Robinson, William H., Blau, Helen M., Steinman, Lawrence
Vydáno v Proc Natl Acad Sci U S A (2018)