A groundbreaking research study co-authored by Dr. Angelica Oviedo, M.D., of Burrell College of Osteopathic Medicine has been accepted for publication in The Journal of Experimental Medicine, offering promising new insight into the treatment of neurodegenerative diseases.
The study, titled “The STING pathway drives non-inflammatory neurodegeneration in NGLY1 deficiency,” demonstrates that neurodegeneration caused by NGLY1 deficiency may be treatable using the STING antagonist VS-X4. NGLY1 deficiency is a rare genetic disorder that impairs protein degradation and cellular stress responses, often leading to severe neurological symptoms.
Dr. Oviedo’s contribution was key in validating that a previously documented human case of NGLY1 deficiency mirrors the animal model used in the current research. Her earlier publication on the systemic and neuropathologic findings of her patient with NGLY1 deficiency led to the current collaboration.
“This research gives us hope that targeted therapies like STING antagonists could be life-changing for patients with NGLY1 deficiency and potentially for those with more common neurodegenerative diseases,” said Dr. Oviedo. “Because NGLY1 deficiency is considered a prototype for broader neurodegenerative processes, the implications extend well beyond this one condition.”
The findings also suggest potential therapeutic benefits for patients with conditions such as Alzheimer and Parkinson disease.
Accepted on May 14, 2025, the manuscript was co-authored by an international team of researchers, including Kun Yang, Gustavo Torres-Ramirez, Nicole Dobbs, Jie Han, Makoto Asahina, Reiko Fujinawa, Angelica Oviedo, Nan Yan, and several others.