Carbohydrate Ligand-Mediated Precise Kidney Delivery of PNA.

Delivering drugs to the specific kidney cells remains a significant challenge in treating renal disorders. This chapter introduces a novel renal tubule targeting carbohydrate (RENTAC) ligand to precisely deliver peptide nucleic acid (PNA) to the proximal convoluted tubules of the kidney. We have reported the synthesis of the RENTAC ligand and a solution-phase PNA conjugation and characterization method. The methodology for elucidating the cellular uptake mechanism in cell culture and comprehensive biodistribution studies and evaluating the PNA-RENTAC conjugate efficacy in kidney fibrosis models is detailed. The reported methods are thoroughly validated and robust. This approach of ligand-mediated precise kidney delivery of PNA opens a vast platform for treating kidney disorders using antisense and antigene technologies.