Title:
Gene Based Neuromodulation
Gene Based Neuromodulation
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Author(s)
Boulis, Nicholas
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Abstract
Nicholas Boulis, M.D. is a Functional Neurosurgeon with significant expertise
in the field of gene transfer to the nervous system. Dr. Boulis' Gene and Cell
Therapy Translational Laboratory pursues advanced biological treatments for
neurological disorders, including Amyotrophic Lateral Sclerosis (ALS, also
known as Lou Gehrig's disease) and Spinal Muscular Atrophy (SMA). Over the last two decades, growing knowledge about the underlying causes of these diseases, 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 antiapoptotic
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 tissuespecific
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.
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Date Issued
2017-03-14
Extent
68:07 minutes
Resource Type
Moving Image
Resource Subtype
Lecture