Zarife Sahenk at Nationwide Children's Hospital and Ohio State University in Columbus, and colleagues, found mice with a disease resembling type 1A Charcot-Marie-Tooth disease (CMT) benefited from a transfer of genes for the neurotrophin 3 protein. CMT1A is caused by a duplication of the PMP22 gene.
Jerry Mendell, who has received many MDA research grants and co-directs the MDA clinic at Nationwide Children's, was part of the study team, as was Brian Kaspar, who has MDA support at Nationwide.
The researchers injected the leg muscles of CMT1A mice with either neurotrophin 3 genes inside shells made from adeno-associated viruses, or with a sham injection. The legs injected with the genes showed better grip strength and had more normal-looking nerve fibers.
The investigators concluded that neutrophin 3 gene therapy is a promising avenue for treatment development in human CMT1A.
These findings were reported at the 2009 American Academy of Neurology meeting, which was held recently in Seattle.