An induced pluripotent stem cell-based chemical genetic approach for studying spinal muscular atrophy
Richard M. Giadone, Kristina M. Holton, Xiaoyu Hu, Ted E. Natoli, Sabrina Ghosh, Stanley Gill, Nicholas Lyons, Aravind Subramanian, Lee L. Rubin
The paper uses a chemical genetic approach with induced pluripotent stem cells to screen for compounds affecting SMA neuronal gene expression, generating a queryable dataset of transcriptional profiles.
Frontiers in Neuroscience · Feb 4, 2026Peer-reviewedNeuroscience