In February 2020, Dr. Kleopa completed milestone 2 of the 24-month project and the results continue to be encouraging. The goal during this 6-12 month period of the project was to further optimize the amount of PMP22 gene silencing in a mouse model of CMT1A. Dr. Kleopa and scientific advisors to the CMT Research Foundation believe that the amount of gene silencing is now adequate to proceed to testing the therapeutic effect in the experimental models..
The next stage of research will be critical to determine if the AAV used and the gene silencing tool are effective in providing a therapeutic benefit in the model of neuropathy, an important milestone that will support future testing in people with CMT1A.
We will keep you apprised of research updates as they happen.