Molecular and Cellular Therapies for Motor Neuron Diseases

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. Dr. Anthony Donsante is a scientist whose research focuses on the development of gene therapies for neurodegenerative diseases. He graduated Magna Cum Laude from Case Western Reserve University with Bachelor of Science degrees in Mathematics and Biology and minors in Artificial Intelligence and History. He went on to earn his Doctorate in Molecular Genetics from Washington University in St. Louis, studying gene therapy for lysosomal storage diseases in the laboratory of Dr. Mark Sands. Dr. Donsante then moved on to a postdoctoral position at the National Institutes of Health, where he developed the first gene therapy for Menkes disease in the laboratory of Dr. Stephen Kaler. Dr. Donsante is currently a Research Associate at Emory University, working with Dr. Nicholas Boulis to develop gene therapies to treat amyotrophic lateral sclerosis.

• 1. Molecular and Extracellular Cues in Motor Neuron Specification and Differentiation2. Natural History of Amyotrophic Lateral Sclerosis3. Genetics of Amyotrophic Lateral Sclerosis4. Molecular Mechanisms of Amyotrophic Lateral Sclerosis5. An Introduction to the Natural History, Genetic Mapping, and Clinical Spectrum of Spinal Muscular Atrophy6. Genetics of Spinal Muscular Atrophy7. Introduction to Gene and Stem-Cell Therapy8. Gene Therapy for Amyotrophic Lateral Sclerosis: Therapeutic Transgenes9. Stem Cell Therapy for Amyotrophic Lateral Sclerosis10. Gene Therapy for Spinal Muscular Atrophy11. Cellular Therapy for Spinal Muscular Atrophy: Pearls and Pitfalls12. Clinical Trials to Date