Collaboration examines nanoparticle drug delivery system in Charcot-Marie Tooth disease

A research collaboration will investigate the use of lipid nanoparticles to deliver therapies for Charcot-Marie-Tooth disease to the peripheral nervous system, according to a press release from the CMT Research Foundation.

“I am excited to work with the CMT Research Foundation as they continue their work on an important, too prevalent disease," James Dahlman, PhD, assistant professor in the Wallace H. Coulter Department of Biomedical Engineering at the Georgia Institute of Technology and Emory School of Medicine, said in the release. “I am hopeful that our approaches to improve gene therapies can lay the foundation for drugs that benefit patients in the future.”

Dahlman and colleagues will research lipid nanoparticles “that can deliver genetic therapies to Schwann cells” with funding from the CMT Research Foundation. The dysfunction of these cells, which protect peripheral nerves, results in disease in the most common form of CMT, according to the release. Dahlman previously developed a system to identify lipid nanoparticles that can enter other types of cells.

Currently, lipid nanoparticles are used to treat another genetic condition and are also the delivery system for two COVID-19 vaccines, according to the release.

If the approach works, findings from this study “could ultimately result in the design of therapies for [patients with CMT] that restore normal protein function,” the CMT Research Foundation said in the release.

Reference:

CMT Research Foundation. CMT Research Foundation launches groundbreaking research to overcome barriers to delivering CMT therapies to the peripheral nervous system. Available at: https://cmtrf.org/dahlman-cmt-research-collaboration-announcement/. Accessed Feb. 17, 2021.