129 research outputs found
Gene Based Neuromodulation
No full textPresented on March 14, 2017 from 8:30 a.m.-9:30 a.m. at the Parker H. Petit Institute for Bioengineering and Bioscience (IBB), room 1128, Georgia Tech.Nicholas Boulis, M.D. is an Associate Professor in the Department of Neurosurgery, Emory University School of Medicine.Runtime: 68:07 minutesNicholas 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
Intraspinal stem cell transplantation for amyotrophic lateral sclerosis: Ready for efficacy clinical trials?
No full textIntraspinal stem cell (SC) transplantation represents a new therapeutic approach for amyotrophic lateral sclerosis (ALS) clinical trials. There are considerable difficulties in designing future efficacy trials, some related to the field of ALS and some that are specific to SCs or the mode of delivery. In October 2015, the most controversial points on SC transplantation were addressed during an international workshop intended to bring together international SC and ALS researchers in a public discussion on a topic for which expertise is limited. During the meeting, a discussion was started on the basic structure of the ideal clinical trial testing the efficacy and safety of SC transplantation. The current document includes a number of consensus points reflecting the design of phase II/III clinical trials
Shifting the balance: cell-based therapeutics as modifiers of the amyotrophic lateral sclerosis–specific neuronal microenvironment
No full text- …
