The PIC-5 Inhibition in ALS

Achedo lab identified this PIC-5 inhibition amongst a candidate that reversed motor neuron death in these patient-specific motor neurons, induced motor neurons. The key here that I think was novel is that they combined the small molecule target approach with bioinformatic analysis and public data sets to identify perturbations similar to transcriptional changes. This rescues not only C9 or 72, the genetic, the cause of ALS, but also sporadic ALS. It rescues neuron survival in both those patient backgrounds; it ameliorates neurogeneration,. neuromuscular junction loss, and motor dysfunction in mice.