Molecular and Cellular Therapies for Motor Neuron Diseases

1. Molecular and Extracellular Cues in Motor Neuron Specification and Differentiation 2. Natural History of Amyotrophic Lateral Sclerosis 3. Genetics of Amyotrophic Lateral Sclerosis 4. Molecular Mechanisms of Amyotrophic Lateral Sclerosis 5. An Introduction to the Natural History, Genetic Mapping, and Clinical Spectrum of Spinal Muscular Atrophy 6. Genetics of Spinal Muscular Atrophy 7. Introduction to Gene and Stem-Cell Therapy 8. Gene Therapy for Amyotrophic Lateral Sclerosis: Therapeutic Transgenes 9. Stem Cell Therapy for Amyotrophic Lateral Sclerosis 10. Gene Therapy for Spinal Muscular Atrophy 11. Cellular Therapy for Spinal Muscular Atrophy: Pearls and Pitfalls 12. Clinical Trials to Date

Dr. Nicholas Boulis is a physician scientist whose research interests include biological neurorestoration and neuromodulation through the use of cell, protein and gene delivery to the nervous system. Dr. Boulis graduated Summa Cum Laude from Yale University with distinction in intensive Biology and Philosophy majors and graduated Magna Cum Laude from Harvard Medical School, winning the Harold Lamport Biomedical Research Award.
Dr. Boulis developed a clinical program focusing on peripheral nerve regeneration, spasticity, pain and Parkinson's Disease, applying advanced microsurgical, radiosurgical and ablative and neural augmentation approaches. He has independently organized and secured funding for an outreach/teaching mission to provide surgical therapy to patients in Guatemala requiring treatment for hydrocephalus and spina bifida. To date, "Project Shunt" has provided over 250 free operations to impoverished Guatemalan children.
Dr. Deirdre O'Connor is a research scientist who has conducted both basic and translational research throughout her scientific career, from undergraduate through to post-doctoral work. She graduated from the National University of Ireland, Galway with a B.Sc. in Biotechnology and a Ph.D. in Developmental Biology. Her research interests have evolved as into a focus on regenerative medicine and gene therapy with a translational application. She is, currently, on developing gene therapies for Amyotrophic Lateral Sclerosis (ALS) and optimizing delivery methods to the CNS. This work is being undertaken with a view to progressing it through the translational pathway and on to clinical trials.