Our Approach

In order to be most effective, gene therapy treatments for neurologic disorders must precisely and accurately deliver a vector carrying a therapeutic gene to the appropriate location in the brain.

Our current work focuses on harnessing specific properties of different viral vectors combined with a specialized neurosurgical delivery platform we have developed to deliver the genetic cargo exactly where it needs to go. This three-part process is how we are developing therapies that can reimagine the future for adults and children living with neurologic diseases.

Conditions we study

Neurodegenerative
- Parkinson’s disease – a progressive brain disorder that causes problems with movement and coordination. There is currently no cure, but treatments are available to manage symptoms.
- Alzheimer’s disease – a brain disorder that worsens over time, affecting memory, thinking skills and, eventually, even the brain’s ability to control bodily functions.
Neurodevelopmental
- Aromatic l-amino acid decarboxylase (AADC) deficiency – caused by mutations in the dopa decarboxylase (DDC) gene that affects the ability of cells in the nervous system to produce dopamine, which is critical to communication between cells. This causes developmental delay, oculogyric crises, weak muscles, problems with coordination of movements, as well as issues with blood pressure, heart rate and body temperature.
- Lysosomal storage disorders – inherited metabolic disorders that cause an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. Different parts of the body can be affected, including the nervous system.
Mitochondrial dysfunction in Neurodegenerative Diseases

The mitochondria – often described as the “powerhouses” of a cell – are essential to cellular function, especially in the brain. In neurodegenerative diseases like Parkinson’s, mitochondria may malfunction and fail to produce the energy needed to properly support brain cell functions or generate products that are damaging to the cell. We are investigating the role mitochondrial health plays in neurodegenerative disease and the therapies that could address it.
Addictions

Meet the team

Faculty and staff

Krystof Bankiewicz, MD, PhD

Lluis Samaranch, PhD

Piotr Hadaczek, PhD
Senior Research Scientist

Jerusha Naidoo, PhD
Research Scientist

Victor Van Laar, PhD
Research Scientist

Meika Travis, MS
Research Associate 1, Laboratory Manager

Allison O’Brien, BS
Program Coordinator, Veterinary Tech

Matthew Rocco, BS
Research Aide

Kierra Dickerson
Office Associate
Students