Exploiting toxin internalization receptors to enhance delivery of proteins to lysosomes for enzyme replacement therapy
S. Sugiman-Marangos, G. L. Beilhartz, X. Zhao, D. Zhou, R. Hua, P. K. Kim, J. M. Rini, B. A. Minassian, R. A. Melnyk
This study creates a chimera of diphtheria toxin and TPP1 to enhance lysosomal enzyme delivery, showing improved uptake and efficacy in a mouse model.
bioRxiv · Jan 22, 2020Pharmacology & Toxicology