Cure SMA has awarded a $75,000 research grant to Chris Lorson, PhD, at the University of Missouri, for his project, “Examining the role of astrocytes and the influence upon lower motor neuron susceptibility in SMA.”
SMA is caused by a mutations in the SMN1 gene. This gene is expressed in every cell in the body; however, not all cell types, or even neurons, become “sick” when SMN levels are very low. The goal of this project is to understand why some neurons get sick while other neurons do not get sick.
Dr. Lorson and his team are more specifically concerned with astrocytes, which are star-shaped cells known to support motor neurons in the brain and spinal cord. They will examine the differences between astrocytes from different regions of the central nervous system, in order to identify new factors that help protect some neurons, while understanding why other neurons are not protected.
Understanding why lower motor neurons become sick and die in SMA, while other neurons do not, will help further our understanding of SMA and provide knowledge which can be used to optimize drug development.
Meet Dr. Lorson
Who are you?
I received my PhD form the University of Missouri in 1997 working on a small DNA virus similar to the AAV vector used in many gene therapy labs these days. I started working in the SMA field in 1997 as a postdoctoral fellow in Dr. Elliot Androphy’s lab at Tufts Medical School. My first lab was at Arizona State University (2000-2002) and I have been at the University of Missouri since 2002.
How did you first become involved with SMA research?
While working in Dr. Androphy’s lab, we found that an important viral protein interacted with the SMN protein. From there, I started looking at the “splicing” of the SMN1 and SMN2 genes, identifying important regulatory regions. My first Cure SMA meeting was in Philadelphia, PA at the airport Marriott in 1998 with a small overhead projector and a slide deck. It must have been a good meeting because nearly everyone in that room is still in the SMA field including Arthur Burghes, Umrao Monani, Livio Pellizzoni, and many more (well not that many, there were only ~20 attendees).
What is your current role in SMA research?
In my lab at the University of Missouri, we have been working on the development of a potent antisense oligonucleotide that significantly extends survival of SMA mice. Additionally, we are examining non-SMN modifiers of disease and collaborating with other SMA labs to develop new SMA drugs.
What do you hope to learn from this research project?
The project objective is to determine why some neurons become “sick” and die in conditions of low SMN protein, while others do not.
How will this project work?
We will use primary co-cultures of motor neurons and astrocytes from both SMA and wild type mice in various combinations to look at the effect of astrocytes, both with low and normal levels of SMN protein, on motor neurons.
What is the significance of your study?
This proposal is designed to investigate why lower motor neurons are specifically impacted in SMA. Understanding why these cells are specifically targeted is important from a biological perspective as well as for designing optimized drugs to treat the complex pathology in SMA.
Basic Research Funding
This grant to Dr. Lorson is part of $1.03 million in new basic research funding that we’re currently announcing.
Basic research is the first step in our comprehensive research model. We fund basic research to investigate the biology and cause of SMA, in order to identify the most effective strategies for drug discovery. We also use this funding to develop tools that facilitate SMA research.