Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal muscular atrophy (SMA) biology. Our Scientific Advisory Board ranks the submitted proposals based on their scientific merit and their alignment with Cure SMA’s research priorities. Funding is then awarded to the highest-ranked projects.
Meet Dr. Jiangbing Zhou, PhD
Jiangbing Zhou, PhD, has been awarded $75,000 for his research project “Non-viral Delivery of Base Editing Therapy for SMA”
Dr. Jiangbing Zhou is the Nixdorff-German Professor of Neurosurgery and Biomedical Engineering at Yale School of Medicine. His research focuses on developing non-viral genetic medicines for the treatment of neurological diseases. He leads a research team working to design new ways of safely delivering genome-editing tools to cells in the brain and spinal cord.
This Cure SMA-funded research project builds on the understanding that SMA is caused by loss of the SMN1 gene and insufficient compensation by the SMN2 gene. Current therapies increase SMN protein levels and have significantly improved outcomes, but they do not fully correct the underlying genetic cause of SMA. Recent studies have shown that a technique called base editing can precisely change a single DNA base in the SMN2 gene. This alters the gene, allowing it to function similar to the missing SMN1 gene, increasing production of functional SMN protein which has improved survival and motor function in mouse models. However, delivering base-editing tools safely and efficiently to motor neurons remains a challenge.
To address this challenge, Dr. Zhou and his team developed the Stimuli-Responsive Traceless Engineering Platform (STEP), a delivery system that uses proteins to transport genome-editing tools to the brain and spinal cord. The proteins clear the body after delivering the genome editors. Preliminary data suggests that STEP can efficiently reach motor neurons in the spinal cord and brainstem, the cells most affected by SMA. In this project, Dr. Zhou will examine if his STEP technology can be utilized in the context of SMA, to deliver base editors to motor neurons. By focusing on a non-viral, potentially one-time gene-editing approach, this research aims to determine whether this is a potentially effective and efficient strategy for lifelong SMN2 conversion.
Glossary:
base editing: a specialized type of genome editing that changes a single DNA letter without cutting both strands of the DNA.
genome-editing: technologies that modify DNA to correct or alter genetic instructions inside cells.
mouse models: a laboratory mouse that has been genetically altered to mimic key aspects of a human disease, allow researchers to study the condition and test treatments.
non-viral genetic medicines: gene-based treatments that use specially designed delivery systems rather than viruses, to carry genetic material into cells.
Cure SMA’s top basic research priorities for 2026 include:
- Enhancing understanding of the molecular, cellular, and biochemical mechanisms that underlie SMA pathology.
- Generating key reagents and tools to facilitate drug development and clinical trials.
- Identifying new therapeutic strategies for treating SMA.
- Identifying drug targets that work synergistically with SMN-upregulating therapeutics to benefit older and symptomatic patients.
In 2026, Cure SMA awarded a total of $750,000 to six scientists to pursue these research objectives!
Thank You!
Special thanks to the Concepcion Family, Nunemaker Family, Weisman Family, Luke 18:1 Foundation, Dhont Foundation, and Cure for Casey 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.