University of Missouri

Chris Lorson

Curators’ Distinguished Professor of Pathobiology and Integrative Biomedical Sciences

Associate Vice Chancellor of Research

Associate Dean of Research for Veterinary Medicine

Christian Lorson, PhD, serves as Associate Vice Chancellor for Research and Strategic Initiatives at Mizzou. He is a Curators’ Distinguished Professor of Pathobiology and Integrative Biomedical Sciences, and is Associate Dean for Research and Graduate Studies in the College of Veterinary Medicine where he has been a professor since 2010. Lorson serves as the director of the Missouri-wide Spinal Cord Injury/Disease Research Program (SCIDRP), a state-wide program designed to fund translational research focused upon spinal cord injuries and neurological disease. Prior to joining MU as a faculty member, Lorson received a BA from Colorado College (1991), a PhD from the Department of Molecular Microbiology and Immunology at the University of Missouri School of Medicine (1997) and was a post-doctoral fellow at Tufts University School of Medicine (2000). He started his faculty career at Arizona State University in the biology department and joined Mizzou in 2002 as an assistant professor.

As a principal investigator in Bond LSC, his laboratory continues to focus upon the molecular genetics and therapeutic development opportunities for neurodegenerative diseases, in particular spinal muscular atrophy (SMA), SMA with respiratory distress type I (SMARD1), and Charcot Marie Tooth (CMT). The laboratory has been supported by grants from NIH, DOD and multiple private foundations (including MDA, CureSMA, CMT Research Foundation), totaling upwards of $40M in external support. Dr. Lorson was the Scientific Director of FightSMA, a patient advocacy group focused on SMA research and governmental advocacy (2005-2017) and is a member of the Muscular Dystrophy Association Research Advisory Committee (2007- present), in addition to reviewing for dozens of agencies worldwide. In 2022, he was elected as a Fellow of the American Association for the Advancement of Science (AAAS). Lorson co-founded Shift Pharmaceuticals in 2017 and is the current Chief Scientific Officer at Shift. Shift has successfully raised $9M in non-dilutive funding from the NIH for SMA and CMT projects, including Phase I SBIRs and a U44 from NIH/NINDS.

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Monique Lorson

Principal Investigator, Bond LSC

Associate Research Professor of Pathobiology and Integrative Biomedical Sciences

Monique Lorson’s research has focused towards understanding how mutations in IGHMBP2 result in two distinct diseases SMARD1 and CMT2S. In collaboration with the C. Lorson laboratory, they have worked on the development of a novel CMT2E mouse model and a vector-based approach to reduce NEFL-mediated disease severity (Perez Lopez et al., 2025; Perez Lopez et al., 2025). She also has active collaborations with Drs. Kamal Singh (IGHMBP2 biochemistry) and Nicole Nichols (respiration).

Towards understanding the role of IGHMBP2 mutations in generating disease, they generated six Ighmbp2 mouse models that are based on SMARD1 and CMT2S patient mutations. Importantly, the Ighmbp2D564N/D564N mouse was the first SMARD1 model published that exhibited the defining clinical symptom of SMARD1, respiratory distress as well as SMARD1 pathology (Smith et al., 2022). Their laboratory also identified the first modifier of SMARD1 disease pathology, ABT1 (Vadla et al., 2023). In this manuscript, they demonstrated that the association of ABT1 with IGHMBP2 significantly increased the ATPase and helicase activity as well as the processivity of IGHMBP2 (changing IGHMBP2 biochemical activity from an inactive to active state).

To understand how a particular mutation impacts molecular function, they examined the six IGHMBP2 mutations in a biochemical context with emphasis on IGHMBP2 activity as an RNA/DNA helicase. They published the biochemical characterization of the IGHMBP2-D565N and IGHMBP2-H924Y mutant proteins (Vadla et al., 2024). For the first time, they demonstrated a correlation between IGHMBP2 biochemical activity associated with the D565N and H924Y mutations and disease severity and pathology in patients and our Ighmbp2 mouse models. Importantly, they show that not all IGHMBP2 mutations are equally amenable to traditional gene therapy applications. The characterization of the Ighmbp2H922Y/H922Y and Ighmbp2D564N/H922Y models was recently published (Ricardez Hernandez et al., 2024). In this manuscript, two important findings were described: 1) early P12 respiratory deficiencies correlate with survival and fitness and 2) motor function deficits are independent of respiratory deficiencies.

Due to the nature of SMARD1 and the phenotypes they have observed in our Ighmbp2 mutant mouse models, they have expanded our studies to include the respiratory circuit, swallowing, the ability to suckle, aspiration and the coordination between respiration and swallowing. These mouse models have provided a valuable resource that has greatly expanded the fields’ knowledge on the function of IGHMBP2 and how IGHMBP2 biochemical function relates to disease development and progression.

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