Abstract
As commonly observed in the intracellular delivery of macromolecules, internalization by cells results in the vast majority of the therapeutic cargo becoming trapped within the endosomal/lysosomal system.1 In this issue of Molecular Therapy, researchers designed a conjugate of N-acetylgalactosamine (GalNAc) and a pH-sensitive, endosome-disrupting polymer that can be co-administered with a small interfering RNA (siRNA) targeted with the same ligand.2 The data demonstrate that maximum silencing of transthyretin expression in hepatocytes is maintained in non-human primates for at least 90 days when the siRNA is co-administered with a targeted polymer to facilitate endosomal escape.
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