Gene Based Neuromodulation - Nicholas Boulis
From Katie Gentilello
Over the last two decades, growing knowledge about the underlying causes of Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig's disease) and Spinal Muscular Atrophy (SMA), as well as the protective effects of special proteins, has given rise to hope for the development of therapies. Dr. Boulis' laboratory specializes in the therapeutic application of the genes for these proteins.
Within the Boulis laboratory, the genes for neural growth factors and anti-apoptotic intracellular proteins are inserted into the DNA of genetically engineered viruses. These viruses, which have been rendered safe through the removal of their native genes, can be used to transfer therapeutic genes into diseased tissue. A variety of vectors are currently being tested in both neuronal cell cultures and in animal models for MND.
In parallel, the Boulis laboratory has focused on the development of tissue-specific targeting strategies. These approaches are designed to deliver molecular therapeutics to an anatomically defined site of interest. Much of this effort has concentrated on motor neuron-specific gene delivery.
Finally, Dr. Boulis has focused on the development of techniques for safe and accurate injection of stem cells into the human spinal cord.
Research in the Boulis laboratory tests basic principles while providing tools for clinical translation. Techniques/assays applied in the lab include: neuronal cell cultures, rodent transgenic colonies, surgery in rodents (mice and rats), locomotor behavior assays in rodents, surgery in large animals (pigs and monkeys), histology, etc.With proof-of-principle in the laboratory and Dr. Boulis' expertise in neurosurgery, the laboratory creates a unique resource for the development and clinical translation of these concepts.