Molecular and Cellular Therapies for Motor Neuron Diseases 1st Edition by Nicholas Boulis, Deirdre Connor, Anthony Donsante 0128025246 9780128025246

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ISBN-10 :  0128025246 

ISBN-13 :  9780128025246

Author: Nicholas M. Boulis, Deirdre O’Connor, Anthony Donsante 

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.

Molecular and Cellular Therapies for Motor Neuron Diseases 1st Table of contents:

Chapter 1. Molecular and Extracellular Cues in Motor Neuron Specification and Differentiation
Abstract
Introduction
Specification of Neuroectoderm
Spinal Cord Patterning
The Motor Neuron Progenitor Domain and Initial Neurogenesis
Molecular Programs in Newborn Motor Neurons
Migration
Motor Neuron Subtypes and Targets
Extracellular Matrix and the Nervous System
Motor Neuron Cell Death
The Glial Switch
Generating Motor Neurons From Pluripotent Stem Cells
Generating Oligodendrocyte Precursor Cells From Pluripotent Stem Cells
Conclusion
References
Chapter 2. Natural History of Amyotrophic Lateral Sclerosis
Abstract
History
Epidemiology
Clinical Presentation
Pathogenesis
Treatments
Summary
References
Chapter 3. Genetics of Amyotrophic Lateral Sclerosis
Abstract
Introduction
Amyotrophic Lateral Sclerosis Genes
Protein Aggregation: Superoxide Dismutase 1
Dysfunction of mRNA Metabolism: TAR DNA-Binding Protein and Fused in Sarcoma
Dysfunction of mRNA and Protein Homeostasis: C9orf72
Beyond C9orf72: Dysfunction of Protein Degradation Systems and the Amyotrophic Lateral Sclerosis–Frontotemporal Dementia Associated Genes
Disruption of Cytoskeleton: PFN1 and TUBA4A
Future Perspectives and Conclusions
References
Chapter 4. Molecular Mechanisms of Amyotrophic Lateral Sclerosis
Abstract
Introduction
RNA Metabolism
Altered RNA Splicing, Transport, and Translation
Adenosine Deaminase Acting on RNA 2
MicroRNAs
Protein Aggregation and Toxicity
Superoxide Dismutase 1
TAR DNA-binding protein-43
Fused in Sarcoma
Other Aggregating Proteins
C9ORF72
Axonopathy and Axonal Transport Defects
Cellular Stress
Stress Granules
Autophagy and Proteasomal Stress
Endoplasmic Reticulum Stress
Inflammation and Glial Function
Astrocytes
Microglia
Oligodendrocytes
T cells
Perineuronal Nets and Extracellular Matrix Dysfunction
Conclusions
References
Chapter 5. An Introduction to the Natural History, Genetic Mapping, and Clinical Spectrum of Spinal Muscular Atrophy
Abstract
Introduction: Epidemiology
Historical Context of Spinal Muscular Atrophy: Review of Case Studies and History of Categorization
Our Modern Understanding of the Disease: Mapping Efforts and Identification of the Survival Motor Neuron Gene
Treatment and Standard of Care
Conclusion
References
Chapter 6. Genetics of Spinal Muscular Atrophy
Abstract
Introduction
The Spinal Muscular Atrophy Gene Region
Genetics of 5q Spinal Muscular Atrophy and Phenotype Modification in Man
The Biochemical Function of Survival Motor Neuron
Spinal Muscular Atrophy Models and Genetic Suppression
Development of Therapies Based on the Genetics of Spinal Muscular Atrophy
Summary and Future Directions
References
Chapter 7. Introduction to Gene and Stem-Cell Therapy
Abstract
Introduction
Gene Therapy
Stem Cells
Routes of Delivery
Conclusion
References
Chapter 8. Gene Therapy for Amyotrophic Lateral Sclerosis: Therapeutic Transgenes
Abstract
Introduction
Antiapoptosis
Glutamate Signaling (Glutamate Transporter 1, ADAR2/AMPA)
Antioxidant Genes
Neurotrophic Factors
Tetanus Toxin heavy Chain C-Fragment
Angiogenin
Single-Chain Antibodies
Possible Future Candidates
Timing of Therapy
Concluding Remarks
References
Chapter 9. Stem Cell Therapy for Amyotrophic Lateral Sclerosis
Abstract
Introduction
Stem Cells in Amyotrophic Lateral Sclerosis: Microenvironment Modulation
Embryonic Stem Cells
Bone Marrow-Derived Mesenchymal Stem Cells
Peripheral Blood Stem Cells
Umbilical Cord Blood Stem Cells
Olfactory Ensheathing Cells
Neural Progenitor Cells
The Future of Stem Cells in Amyotrophic Lateral Sclerosis
Conclusions
Acknowledgments
References
Chapter 10. Gene Therapy for Spinal Muscular Atrophy
Abstract
Introduction
Animal Models
Strategies of Gene Therapy in Spinal Muscular Atrophy
Clinical Trials
Closing Remarks
Acknowledgments
References
Chapter 11. Cellular Therapy for Spinal Muscular Atrophy: Pearls and Pitfalls
Abstract
Introduction
Potential Therapeutic Effects of Stem Cells on Spinal Muscular Atrophy Disease Mechanisms
The Selection of Cell Types to be Transplanted
Embryonic Stem Cell Derived Motor Neuron Precursors for Spinal Muscular Atrophy
Induced Pluripotent Stem Cell Derived Motor Neurons as a Cell Source for Transplantation
Neural Stem Cells
Transplantation of Specific Neural Stem Cell Subpopulations
Glial Cells
Muscle Cells
Minimally Invasive Strategies of Administration to Ease the Clinical Translation: The Cerebrospinal Fluid and Systemic Routes
From Benchtop to Clinical Translation: Issues to Overcome
Stem Cells for Drug Discovery
Conclusions
References
Chapter 12. Clinical Trials to Date
Abstract
Situation and Introduction
Stem Cell Clinical Trials in Amyotrophic Lateral Sclerosis
Europe
Asia
Conclusions

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