Cure SMA has awarded a $140,000 research grant to Christine Beattie, PhD, at the Ohio State University, for her project, “Identification of SMN:HuD bound RNAs critical for motor neuron development.”
Because of a genetic mutation in the SMN1 gene, individuals with SMA don’t produce survival motor neuron protein (SMN protein) at high enough levels, causing motor neurons to stop working properly and eventually die. However, we don’t know all the ways that the loss of SMN protein affects the body.
Several new projects, including Dr. Beattie’s, are looking at these unanswered questions. Dr. Beattie’s project will look specifically at how the loss of SMN protein affects the development of motor neurons, and at other proteins and RNAs that might interact with SMN protein during the development of motor neurons.
Meet. Dr. Beattie
Who are you?
I am a professor at the Ohio State University in the Department of Neuroscience. I first began studying motor neurons during my training at the University of Oregon. Using zebrafish as a model system, we could study and actually see motor neurons in the animal as they developed allowing us to understand how this process was controlled.
How did you first become involved with SMA
When I moved to Ohio State University to start my lab, I was fortunate to meet Dr. Arthur Burghes. He had made some of the key genetic mouse models of SMA and we thought using zebrafish would be a great way to look at motor neurons under conditions of low SMN. With my background, it was a perfect fit! Once I started working on SMN and SMA, I never stopped.
What is your current role in SMA research?
My lab has shown that when SMN levels are low, motor neurons do not develop correctly. We think that this is causing motor neurons to eventually fail. Our goal is to determine the function of SMN in motor neuron development.
What do you hope to learn from this research project?
We have found that motor neurons do not develop properly in a SMA animal model in fish, and we hypothesize that this contributes to the motor neuron dysfunction in this disease. We are investigating how SMN is important for motor neuron development.
How will this project work?
We are looking at proteins and RNAs that SMN interacts with to ask whether they are important for motor neuron development. It is significant that we are looking at these animal models at the time motor neurons are developing as fish embryos, rather than later in development after this process has occurred.
What is the significance of your study?
In SMA, motor neurons fail to function properly leading to muscle paralysis. This project is important because we do not yet understand why motor neurons don’t function correctly when SMN levels are low. If we understood this, we could better design therapeutics, and it would help our overall understanding of what makes motor neurons function correctly.
Basic Research Funding
This grant to Dr. Beattie is part of $890,000 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.
$140,000 to Megerditch Kiledjian, PhD