Dr. Justin Ichida is an AcuraStem co-founder and close collaborator. He is an Associate Professor of Stem Cell Biology and Regenerative Medicine at the University of Southern California Keck School of Medicine and the Director of the Choi Therapeutic Screening Center at the Broad CIRM Center for Stem Cell Research. His major focus is in understanding cellular reprogramming and applying this approach to investigate neurological disorders. Neurodegenerative diseases are driven by diverse and only partially understood pathological stimuli. His lab determined that the forced expression of lineage-specific transcription factors can convert human somatic cells directly into specific neural subtypes that recapitulate bona fide neurodegenerative disease processes in vitro (Son et al, Cell Stem Cell, 2011, Wen et al., Neuron, 2014). They have characterized the resulting human patient-derived neurons and microglia extensively and have identified key molecular pathways that regulate the efficiency and fidelity of reprogramming (Babos et al., Cell Stem Cell 2019). We have used patient-derived neurons to identify haploinsufficiency as a key driving force behind C9ORF72- induced neurodegeneration (Shi et al., Nature Medicine, 2018), and discovered PIKFYVE kinase as a therapeutic target that could address this pathology. The Ichida lab has extensive experience developing AAV and BAC mouse models. For example, they successfully generated human PIKFYVE BAC mice for assessing in vivo ASO-mediated suppression of human PIKFYVE, and successfully established C9ORF72 (AAV)-GR100 mice for assessing ASO efficacy in vivo.