Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in SMA biology. Our Scientific Advisory Board then ranks the submitted proposals on both their scientific merit and relevance to Cure SMA’s research priorities. Funding is awarded to the highest ranked projects.
In 2023, Cure SMA awarded a total of $750,000 to six scientists to pursue our basic research priorities and objectives.
Lyndsay Murray, PhD, at the University of Edinburgh in Scotland was awarded $150,000 for her research project, “Investigating the viability of vulnerable motor axons following SMN restoration in mouse models of SMA.”
Meet Dr. Murray
Dr. Murray leads a research laboratory at the University of Edinburgh in Scotland. She became involved in SMA research during graduate school, when she was one of the first scientists to describe how the connections between motor neurons and muscle break down in SMA mice. After graduate school, Dr. Murray continued to research SMA and in 2014, she established her own lab in Edinburgh.
Today, Dr. Murray’s research group uses SMA mouse models to better understand how motor neurons and muscles recover when treatment is given. They want to find new ways to speed and improve the recovery of motor neurons affected by SMA.
Motor neurons carry messages from the brain to muscles. In people with SMA, deficiency in the survival motor neuron (SMN) protein can cause motor neurons to become unhealthy, causing muscle wasting. Dr. Murray’s group believes that in infants with SMA, a specific sub-group of motor neurons are developmentally immature and vulnerable to disease. They believe that these immature motor neurons do not respond well to currently approved treatments for SMA.
In their current project, Dr. Murray’s group wants to learn more about these immature motor neurons and how to support them. The researchers will use high power microscopes to study immature motor neurons in an inducible SMA mouse model. The inducible SMA mouse is normally deficient in the SMN protein. However, Dr. Murray and her colleagues can use a chemical switch to turn on the expression of SMN in the mouse.
This technique will enable Dr. Murray’s research lab to study how immature motor neurons function with and without the SMN protein. They will also investigate whether a combination of approved SMA treatments can help protect these motor neurons. Insights from this research could improve the effectiveness of current treatments and result in better outcomes for people with SMA.
Cure SMA’s top basic research priorities currently include:
- Learning more about when and where the survival motor neuron (SMN) protein is needed and how it functions in the body.
- Finding new ways to treat SMA, especially those that can be used in combination with approved drugs.
- Using cellular or animal models to better understand the SMA disease process.
- Developing new tools for SMA research, such as new SMA animal models and new ways of tracking disease progression.
Thank You!
Special thanks to the Concepcion Family, Nunemaker Family, Weisman Family, Luke 18:1 Foundation and Dhont Foundation for their generosity to Cure SMA in our quest to invest in basic research that will ultimately drive the next generation of SMA treatments.