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.
Elana Molotsky, PhD, at the Johns Hopkins University School of Medicine was awarded $100,000 for her research project, “Identifying developmental and degenerative mechanisms of SMA using single motor neuron nucleus RNA sequencing.”
Meet Dr. Molotsky
Dr. Elana Molotsky is a post-doctoral fellow in the lab of Dr. Charlotte Sumner at Johns Hopkins University. After completing her doctoral degree studying spinal and bulbar muscular atrophy (SBMA), Dr. Molotsky wanted to continue to research motor neuron diseases. Working with Dr. Sumner on SMA alongside the other motor neuron disease scientists at Johns Hopkins University was an ideal fit for her career goals.
Dr. Molotsky’s current research focuses on finding out why the loss of the SMN protein causes motor neuron death in SMA.
Motor neurons are cells in the central nervous system that relay information from the brain to muscles. In people with SMA, motor neurons can become unhealthy, causing muscles to lose strength and function. Motor neurons contain a chemical messenger called RNA in their nuclei. RNA messages change depending on what is happening inside a motor neuron. For example, the RNA found in a healthy motor neuron will contain different messages than those found in an unhealthy motor neuron.
In her current project, Dr. Molotsky will analyze RNA from the individual spinal cord motor neurons of SMA mice at different stages of disease progression. By determining how RNA messages change as these motor neurons become unhealthy, Dr. Molotsky will learn more about which cellular processes are disrupted in the motor neurons of people with SMA. Her research may also reveal new targets for future SMA drugs.
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.
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.