With CMTA support of $54,051, researchers led by Yizhou Dong, PhD, at the Icahn School of Medicine at Mount Sinai are conducting a study to develop and test new delivery molecules designed to help RNA medicines (a type of genetic medicine) reach nerve cells affected by CMT. Getting medicines to the peripheral nerves remains a major challenge in CMT.
Peripheral nerve cells are protected by specialized biological barriers, such as the blood–nerve barrier, which not only keep out harmful foreign substances but also medicines meant to help. This project focuses on engineering and testing blood–nerve barrier–crossing conjugates (molecules designed to deliver medicines into cells) to help RNA medicines reach Schwann cells.
Dong and his team will use cellular studies in CMT models to assess delivery to peripheral nervous system tissues and to identify the most effective conjugate designs. The goal of this work is to determine whether this delivery strategy is suitable for further development and to generate data that can guide future research aimed at improving RNA-based treatment approaches for CMT.
