A group of US scientists has developed a new way to deliver molecules that target specific genes within cells.
Their platform, which uses a modified form of diphtheria toxin, has been shown to down-regulate critical genes in cancer cells, and could be used for other genetic diseases as well.
The team, led by professors Molly Shoichet and Roman Melnyk of SickKids Hospital, found inspiration from an unexpected source – diphtheria toxin.
Shoichet, said: “A major challenge in the field of drug delivery is most therapeutic vehicles cannot escape the acid environment of the endosome once they get into the cell.
“The diphtheria toxin platform as a delivery vehicle effectively solves that.”
The researchers used the system to deliver molecules that they believed would be effective against glioblastoma.
