A novel cardiotropic AAV variant 4D-C102 demonstrates: superior gene delivery and reduced immunogenicity in cardiac tissues versus wildtype AAV in non-human primates, and results in functional GLA in cardiomyocytes and Fabry fibroblasts

Read More

Kevin Whittlesey, Gabriel Brooks, Roxanne Croze, Chris Schmitt, Paul Szymanski, Julie Nye, Melissa Quezada, Ghezal Beliakoff, David Schaffer, Peter Francis, Melissa Kotterman, David Kirn. Poster presented at the 6th Update on Fabry Disease conference, May 26-28, 2019, Prague, Czech Republic.