AUSTIN, Minn. – A team led by a Hormel Institute researcher is getting $1.8 million to study a rare premature aging disease in children.
The money is coming from the Center for Biologics Evaluation and Research of the Food and Drug Administration and will be given out over a three-year period.
George Aslanidi, PhD, Associate Professor and leader of the Molecular Bioengineering and Cancer Vaccine lab at the Hormel Institute, is leading the research into Cockayne syndrome. There are currently no treatments for the ultra-rare disorder that causes accelerated aging, short stature, microcephaly, photosensitivity, neurological dysfunction, liver and kidney damage, and digestive problems.
“For the next three years, we will work to optimize gene therapy, develop a prototype of cGMP production process, and show that our treatment works in a relevant model. We hope that gene therapy will provide a healthy copy of the affected gene, which will have therapeutic effects resulting in a more positive outcome for patients,” says Dr. Aslanidi.
The other primary investigator in this study is Christina Pacak, PhD, Assistant Professor in the Department of Neurology at the University of Minnesota. Other team members include Peter B. Kang, MD, Director of the Paul and Sheila Wellstone Muscular Dystrophy Center and Kah-Wey Peng, PhD, Director of Toxicology Lab at the Mayo Clinic.
“We hope to collect comprehensive data that support moving forward our developments into a clinical trial, and ultimately, to provide a therapeutic option for patients with Cockayne Syndrome,” says Dr. Aslanidi.