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 2024, Cure SMA awarded a total of $740,000 to six scientists to pursue these objectives!
Rico Schieweck, PhD, has been awarded $149,202 to pursue his research study, “REACT: Rebalancing the Epitranscriptional landscape as an AdvanCed Therapy in SMA.”
Meet Dr. Schieweck
Dr. Schieweck is a Junior Principal Investigator at the Luxembourg Center for Systems Biomedicine (LCSB). He first became involved in SMA research as a postdoctoral fellow when he joined the lab of Dr. Gabriella Viero. Today, his research focuses on how RNA is regulated in brain and spinal cord cells.
After RNA is transcribed from DNA in the cell nucleus, it is modified by the epitranscriptome,” a set of chemical changes to the RNA. These changes can modify the RNA's stability, localization, processing, and/or translation. Dr. Schieweck and his group have observed that the RNA epitranscriptome in cells affected by SMA differs from that of non-SMA affected cells. In this project funded by Cure SMA, Dr. Schieweck and his colleagues will use cells derived from people with SMA to try and understand how loss of the SMN protein contributes to differences in the RNA epitranscriptome.
Findings from this study may help identify new biomarkers for SMA disease progression and treatment response, informing treatment decisions to maximize results for people with SMA.
Cure SMA’s top basic research priorities currently include:
- Learning more about how the survival motor neuron protein (SMN), which is expressed at low levels in SMA, is expressed and functions.
- Understanding the ways the SMN protein affects motor neurons and other cells in the body.
- Developing new treatments and advancing existing drug research, focusing on add-on therapies which work independently of SMN.
- Finding new research tools that can be used to learn more about disease progression and treatment response in SMA.
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.