Charcot Marie Tooth Syndrome (CMT1A)
CMT1A is a hereditary, progressive disease of the peripheral nervous system caused by a duplication in the PMP22 gene. This genetic abnormality causes the overproduction of PMP22 protein in the nervous system, slowly degrading the protective myelination sheaths surrounding sensory and motor axons. Over time, this degeneration causes loss of motor function as well as severe pain and loss of balance. Shift’s lead candidate for CMT1A (compound SHC1A-012) partially blocks the production of PMP22 protein in peripheral nerves, restoring healthy myelin formation, and returning axons and nerves to a property functioning state.
Wild Type Mouse
Genetically modified to develop CMT, untreated
Genetically modified mouse, treated with Shift Molecule