Molecular and Cellular Therapies for Motor Neuron Diseases

Molecular and Cellular Therapies for Motor Neuron Diseases discusses the basics of the diseases, also covering advances in research and clinical trials. The book provides a resource for students that will help them learn the basics in a detailed manner that is required for scientists and clinicians.

Users will find a comprehensive overview of the background of Amyotrophic Lateral Sclerosis (ALS/Lou Gehrig?s Disease) and Spinal Muscular Atrophy (SMA), along with the current understanding of their genetics and mechanisms. In addition, the book details gene and cell therapies that have been developed and their translation to clinical trials.

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

Advanced graduate students, researchers, and clinicians in the fields of neuroscience, neurology, and gene and cell therapy

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

• Provides an overview of gene and cell therapies for amyotrophic lateral sclerosis (ALS) and other motor neuron diseases
• Edited by a leading Neurosurgeon and two research scientists to promote synthesis between basic neuroscience and clinical relevance
• Presents a great resource for researchers and practitioners in neuroscience, neurology, and gene and cell therapy