With CMTA support of $66,000, researchers led by Jiangbing Zhou, PhD, at Yale University are conducting a study to evaluate a new non-viral delivery system called STEP. This system is designed to transport genome-editing medicines to peripheral nerve cells. Getting medicines to the right cells remains a central 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 will test whether the delivery system can carry genome-editing medicines to where they are needed, including Schwann cells and sensory neurons in CMT models.
Zhou and his team will study how well the system reaches peripheral nerve tissues following different routes of administration and whether genome-editing activity can be detected in the targeted cells.
June 2026 Update
Zhou’s lab has produced encouraging early results, demonstrating that STEP can deliver genome-editing tools to peripheral nerve cells. The researchers are now evaluating the platform’s performance and safety while refining the formulation to identify the version best suited for delivering future genome-editing medicines. If successful, this work could help advance a delivery approach for one-time genetic treatments for people living with CMT.
The goal of this work is to determine whether this delivery approach is suitable for further development in CMT research and to generate data that can guide future studies focused on genome-editing strategies in CMT.
