Therapeutic strategies for CMT and inherited neuropathies

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Neuromolecular Med. 2006;8(1-2):255-78.

Therapeutic strategies for the inherited neuropathies.

Shy ME.

Department of Neurology and Center for Molecular Medicine and

Genetics, Wayne State University School of Medicine, Detroit MI

48201, USA.

More than 30 genetic causes have been identified for the inherited

neuropathies collectively referred to as Charcot-Marie-Tooth (CMT)

disease. Previous therapies for CMT were limited to traditional

approaches such as rehabilitation medicine, ambulation aids, and pain

management.

Identification of the genes causing CMT has led to improved genetic

counseling and assistance in family planning.

Identification of these genes is beginning to delineate common

molecular pathways in multiple forms of CMT that can be exploited in

future molecular therapies.

Scientifically based clinical trials for CMT are currently being

implemented. Techniques of gene therapy are advancing to the point

that they may become feasible options for patients with CMT and other

neurodegenerative diseases.