New Collaboration in Charcot-Marie-Tooth Disease to Uncover Ways to Deliver Gene Therapies

James Dahlman, a scientist and professor at both Emory University and the Georgia Institute of Technology, has partnered with the CMT Research Foundation. The aim of this partnership is to identify lipid nanoparticles which could be used as vehicles for moving gene therapies into the cells necessary to combat Charcot-Marie-Tooth disease (CMT).

This is a current major obstacle in the development of gene therapies for CMT, and this partnership could bring real benefits to patients.

The Process

CMT impacts the peripheral nervous system in the body, which is very complex and very delicate.

The nerves are crucial for many of the body’s vital processes as they transmit messages from the brain and spinal cord to the body. A selective barrier (called the blood-nerve barrier) around nerve cells protects them from viruses and other harmful agents, therefore protecting the crucial system. However, these very barriers which protect the nerves can also prevent vital therapies, such as gene therapy, from reaching the cells they must reach to be effective.

Researchers understand that gene therapies can reach the cells when they are encased in a lipid nanoparticle. This is because the lipid nanoparticles are quite similar to other fat molecules within the body. The body recognizes them as fat molecules and therefore, allows them to enter the cells.

Dahlman discovered a way to effectively screen the nanoparticles to identify the ones that have the capacity to permeate the cells. Most importantly, his investigation includes cells of the peripheral nervous system. He has already developed gene therapy vehicles for cells in the liver and has many others in the work.

Specifically for this new collaboration, Dahlman will investigate nanoparticles that can permeate Schwann cells which create the myelin sheath protecting the nerve cells.

You can read more about this new collaboration and what it could lead to for CMT patients here.

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