Pharmacology & ToxicologybioRxiv
Exploiting toxin internalization receptors to enhance delivery of proteins to lysosomes for enzyme replacement therapy
S. Sugiman-Marangos, G. L. Beilhartz, X. Zhao…·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.
DOI 10.1126/sciadv.abb0385