Does GM6 reduce C9orf72?
GM604 reduced the expression of C9orf72 by 47% in 2 hours.
The search for a compound to modulate C9orf72, a single target gene will not get out of the lab or into clinical trials for a long time. More importantly fixing one gene will not cure ALS.
Mutations or over-expression in C9orf72 have been found to cause ALS. The protein that is produced may be altered and may interfere with cell function. Some ALS patients also develop frontotemporal dementia.
In a PCR array designed to measure the effect of GM604 on C9orf72, a neuronal SHSY5Y cell line was cultured with GM604,resulting in a decrease in C9orf72 of 47% in 2 hours.