An AAV capsid reprogrammed to bind human Transferrin Receptor mediates brain-wide gene delivery
Qin Huang, Ken Y. Chan, Jason Hongliu Wu, Nuria Roxana Botticello-Romero, Qingxia Zheng, Shan Lou, Casey Keyes, Alexander Svanbergsson, Jencilin Johnston, Allan Mills, C.-L. Lin, Pamela P. Brauer, Gabrielle Clouse, Simon Pacouret, John W. Harvey, , Benjamin E. Deverman
The paper develops a modified AAV capsid that targets the human Transferrin Receptor to enhance gene delivery to the CNS, showing improved reporter expression and therapeutic potential for Gaucher disease.
Science · May 16, 2024Peer-reviewedNeuroscience