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, which are a type of CMT gene therapy, 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.
April 2026 Update
In the first six months, the team built and verified one of the lead delivery molecules in this study, designed to carry RNA medicines across the blood-nerve barrier (biological barriers that shield peripheral nerves from the outside world, including medicines meant to help). Confirming that this molecule was built correctly and holds together stably is a critical first step before any testing in living cells can begin.
With these materials ready, Dong’s lab will now test whether the delivery molecules can successfully carry RNA medicines into neurons and Schwann cells, the two cell types most directly damaged by CMT. These tests will begin in cell culture and advance to CMT models.
CMT damages the nerves that let you walk, use your hands, and feel the ground under your feet, and that damage builds up over time and does not heal. Researchers have medicines that may be able to slow or stop that damage, but right now, there is no reliable way to get those medicines to the nerves that need them. That is the problem Dr. Dong and his lab are working to solve.
If we can deliver CMT gene therapies to these nerves, we might be able to stop the damage before it happens. And damage that never happens means function you never lose.
