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Molecular and Cellular Therapies for Motor Neuron Diseases [Kõva köide]

Edited by (Department of Neurosurgery, Emory University, Atlanta, GA, USA), Edited by (Department of Neurosurgery, Emory University, Atlanta, GA, USA), Edited by (Department of Neurosurgery, Emory University, Atlanta, GA, USA)
  • Formaat: Hardback, 336 pages, kõrgus x laius: 229x152 mm, kaal: 630 g
  • Ilmumisaeg: 16-Jan-2017
  • Kirjastus: Academic Press Inc
  • ISBN-10: 0128022574
  • ISBN-13: 9780128022573
Teised raamatud teemal:
Molecular and Cellular Therapies for Motor Neuron DiseasesSuurem pilt
  • Formaat: Hardback, 336 pages, kõrgus x laius: 229x152 mm, kaal: 630 g
  • Ilmumisaeg: 16-Jan-2017
  • Kirjastus: Academic Press Inc
  • ISBN-10: 0128022574
  • ISBN-13: 9780128022573
Teised raamatud teemal:
Püsilink: https://www.kriso.ee/db/9780128022573.html
Märksõnad:

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.

  • 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

Muu info

A comprehensive overview of gene and cell therapies for ALS and SMA that have been developed and translated into clinical use
List of Contributors
xi
Acknowledgment xiii
1 Molecular and Extracellular Cues in Motor Neuron Specification and Differentiation
R.L. Swetenburg
S.L. Stice
L. Karumbaiah
Introduction
2(1)
Specification of Neuroectoderm
3(1)
Spinal Cord Patterning
3(2)
The Motor Neuron Progenitor Domain and Initial Neurogenesis
5(1)
Molecular Programs in Newborn Motor Neurons
5(1)
Migration
6(1)
Motor Neuron Subtypes and Targets
6(4)
Extracellular Matrix and the Nervous System
10(3)
Motor Neuron Cell Death
13(1)
The Glial Switch
13(1)
Generating Motor Neurons From Pluripotent Stem Cells
14(3)
Generating Oligodendrocyte Precursor Cells From Pluripotent Stem Cells
17(1)
Conclusion
17(1)
References
18(7)
2 Natural History of Amyotrophic Lateral Sclerosis
M.J. Magnussen
J.D. Glass
History
25(1)
Epidemiology
26(3)
Clinical Presentation
29(5)
Pathogenesis
34(1)
Treatments
35(1)
Summary
36(1)
References
37(7)
3 Genetics of Amyotrophic Lateral Sclerosis
K.P. Kenna
J.E. Landers
N. Ticozzi
Introduction
44(1)
Amyotrophic Lateral Sclerosis Genes
45(3)
Protein Aggregation: Superoxide Dismutase 1
48(1)
Dysfunction of mRNA Metabolism: TAR DNA-Binding Protein and Fused in Sarcoma
49(2)
Dysfunction of mRNA and Protein Homeostasis: C9orf72
51(1)
Beyond C9orf72: Dysfunction of Protein Degradation Systems and the Amyotrophic Lateral Sclerosis--Frontotemporal Dementia Associated Genes
52(1)
Disruption of Cytoskeleton: PFN1 and TUBA4A
53(2)
Future Perspectives and Conclusions
55(1)
References
55(7)
4 Molecular Mechanisms of Amyotrophic Lateral Sclerosis
M. Collins
R. Bowser
Introduction
62(1)
RNA Metabolism
63(1)
Altered RNA Splicing, Transport, and Translation
64(2)
Adenosine Deaminase Acting on RNA 2
66(1)
MicroRNAs
66(1)
Protein Aggregation and Toxicity
67(2)
Superoxide Dismutase 1
69(1)
TAR DNA-Binding Protein-43
70(1)
Fused in Sarcoma
71(1)
Other Aggregating Proteins
72(1)
C90RF72
72(2)
Axonopathy and Axonal Transport Defects
74(2)
Cellular Stress
76(1)
Stress Granules
76(1)
Autophagy and Proteasomal Stress
77(2)
Endoplasmic Reticulum Stress
79(1)
Inflammation and Glial Function
80(2)
Astrocytes
82(1)
Microglia
83(1)
Oligodendrocytes
84(1)
T Cells
85(1)
Perineuronal Nets and Extracellular Matrix Dysfunction
86(1)
Conclusions
87(1)
References
88(14)
5 An Introduction to the Natural History, Genetic Mapping, and Clinical Spectrum of Spinal Muscular Atrophy
A. McDonough
L. Urquia
N. Boulis
Introduction: Epidemiology
102(1)
Historical Context of Spinal Muscular Atrophy: Review of Case Studies and History of Categorization
102(9)
Our Modern Understanding of the Disease: Mapping Efforts and Identification of the Survival Motor Neuron Gene
111(2)
Treatment and Standard of Care
113(2)
Conclusion
115(1)
References
116(5)
6 Genetics of Spinal Muscular Atrophy
A.H.M. Burghes
V.L. McGovern
Introduction
121(1)
The Spinal Muscular Atrophy Gene Region
122(2)
Genetics of 5q Spinal Muscular Atrophy and Phenotype Modification in Man
124(5)
The Biochemical Function of Survival Motor Neuron
129(1)
Spinal Muscular Atrophy Models and Genetic Suppression
130(2)
Development of Therapies Based on the Genetics of Spinal Muscular Atrophy
132(2)
Summary and Future Directions
134(1)
References
135(6)
7 Introduction to Gene and Stem-Cell Therapy
D.M. O'Connor
Introduction
141(1)
Gene Therapy
142(7)
Stem Cells
149(6)
Routes of Delivery
155(1)
Conclusion
156(1)
References
157(11)
8 Gene Therapy for Amyotrophic Lateral Sclerosis: Therapeutic Transgenes
A. Donsante
Introduction
168(1)
Antiapoptosis
168(4)
Glutamate Signaling (Glutamate Transporter 1, ADAR2/AMPA)
172(3)
Antioxidant Genes
175(2)
Neurotrophic Factors
177(13)
Tetanus Toxin Heavy Chain C-Fragment
190(1)
Angiogenin
191(1)
Single-Chain Antibodies
191(1)
Possible Future Candidates
192(1)
Timing of Therapy
193(1)
Concluding Remarks
194(1)
References
194(14)
9 Stem Cell Therapy for Amyotrophic Lateral Sclerosis
K.S. Chen
E.L. Feldman
Introduction
208(1)
Stem Cells in Amyotrophic Lateral Sclerosis: Microenvironment Modulation
209(2)
Embryonic Stem Cells
211(1)
Bone Marrow-Derived Mesenchymal Stem Cells
212(2)
Peripheral Blood Stem Cells
214(1)
Umbilical Cord Blood Stem Cells
215(1)
Olfactory Ensheathing Cells
216(1)
Neural Progenitor Cells
217(5)
The Future of Stem Cells in Amyotrophic Lateral Sclerosis
222(1)
Conclusions
223(1)
Acknowledgments
223(1)
References
223(10)
10 Gene Therapy for Spinal Muscular Atrophy
M.R. Miller
E.Y. Osman
C.L. Lorson
Introduction
233(1)
Animal Models
234(2)
Strategies of Gene Therapy in Spinal Muscular Atrophy
236(8)
Clinical Trials
244(2)
Closing Remarks
246(1)
Acknowledgments
246(1)
References
246(6)
11 Cellular Therapy for Spinal Muscular Atrophy: Pearls and Pitfalls
I. Faravelli
S. Corti
Introduction
252(1)
Potential Therapeutic Effects of Stem Cells on Spinal Muscular Atrophy Disease Mechanisms
253(2)
The Selection of Cell Types to Be Transplanted
255(1)
Embryonic Stem Cell Derived Motor Neuron Precursors for Spinal Muscular Atrophy
256(1)
Induced Pluripotent Stem Cell Derived Motor Neurons as a Cell Source for Transplantation
256(3)
Neural Stem Cells
259(1)
Transplantation of Specific Neural Stem Cell Subpopulations
260(1)
Glial Cells
261(1)
Muscle Cells
262(1)
Minimally Invasive Strategies of Administration to Ease the Clinical Translation: The Cerebrospinal Fluid and Systemic Routes
263(2)
From Benchtop to Clinical Translation: Issues to Overcome
265(2)
Stem Cells for Drug Discovery
267(2)
Conclusions
269(1)
References
270(8)
12 Clinical Trials to Date
B.J. Mader
N. Boulis
Situation and Introduction
278(6)
Stem Cell Clinical Trials in Amyotrophic Lateral Sclerosis
284(9)
Europe
293(5)
Asia
298(6)
Conclusions
304(1)
References
305(4)
Index 309
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 OConnor 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.