Bankiewicz Laboratory
Dr. Krystof Bankiewicz’s laboratory is a translational neuroscience group. They have developed several clinical programs based on MR-guided delivery of gene therapy and their translational efforts led to the initiation of four clinical trials in different neurological disorders (Parkinson’s disease and AADC deficiency) and brain tumors (brain stem glioma and glioblastoma). Currently, they are also developing new treatments for adult and pediatric neurologic and neurodevelopmental disorders such as Alzheimer’s disease, Huntington’s disease, multiple system atrophy and lysosomal storage disorders as well as for substance abuse disorders. As a part of the overall gene therapy platform, they’ve also developed devices and procedures to advance intracranial AAV-based gene therapies, cell therapies and drug delivery for neurological disorders and brain cancer for first-in-human clinical trials.
As a Research Scientist in the Bankiewicz Lab, Dr. Naidoo’s research is focused on developing novel gene therapies for sensorineural hearing loss, the most common sensory disorder in humans. Specifically, she develops gene therapies targeted to auditory nerves for the treatment of hearing loss due to noise exposure, infection, and ototoxic drug exposure. The majority of gene therapy approaches for sensorineural neural hearing loss have focused primarily on gene delivery to the hair cells (the sensory cells in the ear). Although the inner ear is typically considered to be a closed system – the health and integrity of spiral ganglion neurons are also important to maintain to allow perception of sound.
Dr. Naidoo undertook her postdoctoral training under the supervision of Dr. Krystof Bankiewicz, a leader in the gene therapy field in the translational and clinical development gene therapies for neurological disorders. Dr. Naidoo collaborates with Dr. Yin Ren (Department of Otolaryngology) and Dr. Eric Bielefeld (Department of Speech and Hearing Science).
As a Research Scientist in the Bankiewicz Lab, Dr. Victor Van Laar’s research focuses on understanding the role of mitochondrial dysfunction, cellular bioenergetics, and neuronal homeostasis in neurodegenerative diseases and other neurologically related disorders, including Parkinson’s disease, substance abuse, and depression. His research utilizes rodent models to better understand why these neurological conditions develop and how they might be treated. By developing and utilizing novel approaches for the delivery of therapeutic agents to target specific regions in the brain, Dr. Van Laar is also exploring gene therapy approaches to treating these brain disorders. Gene transfer and gene editing therapeutic approaches in the brain are evaluated by a combination of behavioral assessments and biochemical assays to evaluate efficacy. Techniques including immunohistochemistry, microscopy, quantitative image analyses, HPLC, and protein chemistry are utilized to assess the translational relevance of potential gene therapies.
Herson Laboratory
Dr. Herson’s research focuses on understanding mechanisms of injury and repair following ischemic brain injury, with studies related to ion channels/receptors, neuroinflammation, age and gender. Current studies are focused on the impact of ischemia on synaptic function and plasticity with the goal of revealing pharmacological interventions that both prevent acute ischemic injury and improve long-term brain function after injury. He received several NIH, DoD and AHA grants and has been continuously NIH funded throughout his independent career. Dr. Herson has published over 100 peer reviewer articles, developed multiple patents and mentored several junior faculty, both PhD and clinician-scientists.
Kolb Laboratory
The Kolb Lab is devoted to the understanding of molecular pathways that, when altered, result in diseases of the motor neuron. They are particularly interested in alterations in RNA metabolism that result in neurological diseases. The mechanisms of spinal muscular atrophy and of distal hereditary neuropathies are current foci for biochemical and cell-based investigation. Their long-term goals are to determine the precise mechanisms that cause motor neuron diseases, including sporadic amyotrophic lateral sclerosis, and to develop small molecule and/or gene-based therapies for these diseases.
Orfila Laboratory
The Orfila laboratory focuses on understanding mechanisms of injury and repair following Traumatic brain injury (TBI), a leading cause of mortality and morbidity in adults, with significant sequelae including memory deficits. Despite intense research, no pharmacological interventions are currently available to improve functional recovery following TBI. The Orfila lab currently investigates the effects of TBI on memory acquisition and retrieval in a gender and age dependent manner. Through electrophysiological, behavioral and molecular methods, these studies focus on investigating the important cellular mechanisms underlying impaired hippocampal function, an area known for memory storage and retrieval. To advance their findings from mechanism to translation, they are using viral-mediated gene therapy as therapeutic strategies targeting pathways identified in their basic animal studies.
Samaranch Laboratory
Lluis Samaranch, Ph.D., is an Assistant Professor in the Neurologic Surgery department and the Gene Therapy Institute at The Ohio State University. His laboratory focuses on the development of AAV-based gene therapies for inherited disorders, including Niemann-Pick Type A disease, MAO-A deficiency, Parkinson’s disease, and Alzheimer’s disease among other neurological disorders. His research goals are also to characterize the axonal transport of new capsid-engineered vectors for in vivo gene transfer applications to the CNS, the immunological consequences of the AAV-mediated gene transfer into the CNS, and the development of new surgical approaches to improve gene delivery into the CNS, including direct brain infusion, CSF injection and in utero gene transfer.
Townsend Laboratory
The Townsend Lab for Neurobiology & Energy Balance investigates how the nervous system (brain and peripheral nerves) regulate appetite, energy expenditure, and metabolic health. To do this, we take a broad physiological approach ranging from basic neurobiology to translational/clinical experimentation, encompassing neuroscience, cell and molecular biology, biochemistry, and metabolic physiology approaches using cell culture, mouse transgenic models, and human tissue samples. Lab projects fall in two areas of focus: 1) brain/hypothalamic adult stem cells and neural plasticity in the regulation of energy balance homeostasis; and 2) brain-adipose neural communication and adipose innervation by sensory and sympathetic nerves, including neurovascular and neuroimmune interactions, neurotrophic factor action, and adipose neural plasticity and neuropathy. Our research program impacts the study of obesity, diabetes, cardiovascular disease, cancer, and aging. Gene therapy projects include targeting peripheral nerves, adipose tissue, and the hypothalamus in the improvement of metabolic health and neural plasticity/remodeling.
As a Research Scientist in the Townsend lab, Dr. Magdalena Blaszkiewicz’s research program focuses on adipose neuropathy and potential gene therapy treatments, as well as neuroimmune interactions in the maintenance and control of adipose axonal health and plasticity. Her future independent research will focus on investigating how lymphatic vessels and lymph nodes in adipose serve as a nexus of neuroimmune cell crosstalk. A goal of this research is to determine if and how loss of nerves around lymphatic vasculature and lymph nodes in adipose tissues affect immune cell functions and trafficking, as well as fluid homeostasis in health and disease.