Bio:

Dr. Lorson obtained his PhD in 1997 from the University of Missouri, School of Medicine in the Department of Molecular Microbiology and Immunology under the advisement of Dr. David Pintel. Dr. Lorson joined Dr. Elliot Androphy at the New England Medical Center, Tufts University in Boston, MA from 1997-2000 where he as a post-doctoral fellow and Assistant Research Professor before accepting an Assistant Professor position at Arizona State University. In 2002, Dr. Lorson joined the faculty at the University of Missouri with a dual appointment in the College of Veterinary Medicine and the College of Medicine (Veterinary Pathobiology, and Molecular Microbiology, respectively). Dr. Lorson advanced to Full Professor in 2010 and has served as the Associate Chair for the Department and currently functions as the Associate Dean for Research and Graduate Studies for the CVM. From 2005-2017, Dr. Lorson served as the Scientific Director of FightSMA, a non-profit focused upon SMA research and advocacy, and served as the Co-Chair of the Scientific Advisory Committee. Dr. Lorson is currently a member of the Bond Life Sciences Center at the University of Missouri. In 2017, Shift Pharmaceuticals was co-founded by Drs. Lorson and Steve O’Connor.

Dr. Lorson’s research has focused primarily upon neurodegenerative diseases including Spinal Muscular Atrophy (SMA) for nearly 20 years. Originally, the lab’s work detailed the molecular mechanisms that regulated pre-mRNA splicing of a critical exon, including the identification of the “C/T” distinction between SMN1 and SMN2 as well as cis- and trans-factors that regulate SMN exon 7 inclusion/exclusion. Eventually, the lab’s research evolved into translational studies designed to develop therapeutics, including developing novel animal models and technologies that re-direct the pathogenic SMN2 splicing event that is central to SMA development. Based upon these results, similar approaches are now being investigated in other neurodegenerative disorders.

Abstract:

Therapeutic Strategies for Spinal Muscular Atrophy

Spinal Muscular Atrophy (SMA) is the leading genetic cause of infantile death worldwide. SMA is caused by homozygous loss of the SMN1. With the recent clinical success of SMA therapeutics and the FDA approval of two novel therapeutics, the path towards second-generation compounds has been laid out by these early successes. To address the breadth and complexity of the SMA patient population, additional therapeutics are needed. To this end, we are examining a targeted series of SMA modifying genes, including Stathmin-1, to provide biological understanding and to develop novel therapeutic targets. The discovery of new pathways in combination with SMN-dependent strategies will hopefully begin to address the needs of all SMA patients.