One year after undergoing a novel gene therapy procedure to treat a rare genetic neurological disorder at The Ohio State University Wexner Medical Center, Delilah Ramirez was taking steps forward. Since then she has continued to progress, walking unassisted and reaching new milestones once believed to be impossible.

Every step she watches her daughter, Delilah, take is a miracle to Arcelia Ramirez. Delilah underwent a novel approach to gene therapy to treat a rare genetic disorder at The Ohio State University Wexner Medical Center in July 2022. Now she continues to surprise her mom with her progress, including walking on her own.

Delilah Ramirez, 9, is now able to feed herself after receiving treatment for a rare genetic disorder that causes severe physical and cognitive disability. Tasks like these were once believed to be impossible for Delilah. But now she can handle them after an experimental procedure at The Ohio State University Wexner Medical Center that delivered gene therapy directly to her brain.

Krystof Bankiewicz, MD, PhD,collaborates with his team in the lab at The Ohio State University Wexner Medical Center’s Gene Therapy Institute. Bankiewicz and others developed the technology to deliver therapeutics directly to the precise area of the brain affected by different genetic and neurological disorders.

Using real-time MRI allows neurosurgeons at The Ohio State University Wexner Medical Center to precisely deliver a genetic therapeutic (white circle) directly to the precise site of the brain (outlined in green) to help patients with a rare genetic disorder called AADC deficiency. This novel approach to gene therapy is also used in clinical trials at Ohio State to treat a variety of genetic disorders, along with more common neurological diseases including Alzheimer’s and Parkinson’s disease.

Krystof Bankiewicz, MD, PhD, chief scientific officer of the Gene Therapy Institute at The Ohio State University and professor of Neurological Surgery at The Ohio State University Wexner Medical Center and College of Medicine.

J. Bradley Elder, MD, associate professor in the Department of Neurological Surgery at The Ohio State University and director of Neurosurgical Oncology at The Ohio State University Comprehensive Cancer Center—Arthur G. James Cancer Hospital and Richard J. Solove Research Institute