Rate My Professor Chris Lorson

The research in the Chris and Monique Lorson Laboratory centers on translational medicine, molecular genetics, and gene therapy strategies for neurodegenerative disorders, with a primary emphasis on spinal muscular atrophy (SMA), SMA with respiratory distress type 1 (SMARD1), and Charcot-Marie-Tooth disease (CMT). The lab has pioneered developments such as a splice-switching antisense oligonucleotide that significantly extends survival in SMA mouse models from a single neonatal dose, and has generated novel animal models for SMARD1 and CMT2S. Supported by more than $40 million in extramural funding from the NIH, Department of Defense, and organizations including the Muscular Dystrophy Association, CureSMA, and CMT Research Foundation, the lab collaborates on small molecule therapeutics and AAV-mediated gene delivery. Lorson co-founded Shift Pharmaceuticals in 2017, serving as Chief Scientific Officer; the company has obtained $9 million in non-dilutive NIH funding, including Phase I SBIR grants and a U44 award from NINDS. His contributions have earned him election as a Fellow of the American Association for the Advancement of Science in 2022. Select publications include “ABT1 modifies SMARD1 pathology via interactions with IGHMBP2 and stimulation of ATPase and helicase activity” (JCI Insight, 2023), “Potential therapeutic targets for Mpox: the evidence to date” (Expert Opin Ther Targets, 2023), “Motor unit recovery following Smn restoration in mouse models of spinal muscular atrophy” (Hum Mol Genet, 2022), and “A combinatorial approach increases SMN level in SMA model mice” (Hum Mol Genet, 2022).