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Journeys In Medicine And Research On Three Continents Over 50 Years

Journeys In Medicine And Research On Three Continents Over 50 Years

Smith Moyra

(2017)

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Table of Contents

Section Title Page Action Price
Contents xi
Epigraph vii
Preface ix
Acknowledgements xv
Part I Social Dimensions of Health 1
1. Looking Back 3
The Witwatersrand and Mining 4
Political Tensions 5
2. Socio-economic Factors and Health: Medical School and Beyond 7
3. Trauma and Experiences in the Surgical Unit at Baragwanath 9
Esophageal Cancer 14
4. South Africa in the Past and in the Present Millennium 17
WHO Millennium Development Goals (MDG) 19
References 19
Part II Mothers and Children 21
5. Pregnancy and Hazards 23
Pre-eclampsia and Eclampsia 24
Uterine Rupture 25
Maternal Morbidity and Mortality during Labor and Delivery and in the Post-partum Period 26
Measures to Reduce Maternal Mortality and Morbidity 27
6. Newborn Infants: Prematurity and Other Problems 29
Prematurity and Causes 31
Preterm Birth in Developed Countries 32
Spontaneous Preterm Birth 32
Preterm Births in the USA 33
Survival Rates of Preterm Infants 33
7. Neural Tube Defects 35
World-wide Studies on Neural Tube Defects 38
Pathogenesis of Neural Tube Defects 38
Measurable Markers of Neural Tube Defectsduring Pregnancy 39
8. Thyroid Hormone Deficiency in Infants and Children 41
Historical Discoveries Related to Hypothyroidism 42
Hypothalamic–pituitary Axis and Thyroid Function 44
Iodine Deficiency in Recent Decades 45
References 46
Part III Infection Related Diseases 51
9. Fading, Emerging and Re-emerging Infectious Diseases 53
Rheumatic Fever 55
Damage to Heart Valves and Further Complications 57
Bacterial Endocarditis in the 21st Century 57
Post-streptococcal Glomerulonephritis 58
Prevention of Rheumatic Heart Disease and Acute Glomerulonephritis in the Antibiotic Era 58
Invasive Infections: New Diseases or Newly Recognized Diseases? 59
10. Tuberculosis 61
A History of Tuberculosis 63
Tuberculosis and Societal Conditions 64
Tuberculosis Returns 65
Tuberculosis and the Possibilities for Effective Vaccines 65
Biomarkers of Active Tuberculosis 67
11. Tetanus 69
12. Viral Infections that May Lead to Cancer 73
(a) Hepatitis Viruses and Hepatoma 73
Discovery of Hepatitis B Virus 74
Hepatitis B Virus and Hepatoma 75
Development of an HBV Vaccine 75
Successes Achieved with HBV Vaccine 76
Hepatitis B Worldwide 76
Hepatitis B and Hepatitis C in Hepatocellular Carcinoma 77
Hepatocellular Carcinoma and Hepatitis B and C Viruses Worldwide 77
Attempts to Control Hepatitis C Virus Infections 77
Mycotoxins 78
(b) Human Papilloma Virus, Cervical and other Cancers 78
Development of a Vaccine that Induces Immunity to Human Papilloma Viruses 79
(b) Herpes 8 Virus and Kaposi Sarcoma 79
13. HIV/AIDS 81
References 81
Part IV Infections, Therapy and Prevention 87
14. The Golden Age of Antibiotics, and Has it Passed? 89
The Golden Age 89
A History of Penicillin Discovery 91
Antibiotic Discovery Processes 92
15. Antibiotic Resistance Mechanisms 95
Genomes, Mobilomes, Information Transfer — Another Mechanism of Antibiotic Resistance Acquisition 96
Salmonella enterica Variants and Plasmids 97
Drug Resistant Mycobacterium tuberculosis 98
16. Genomics and Natural Product Discovery 101
Additional Methods for Discovery of New Antibiotics 102
New Methods to Identify “Unculturable” Soil Microbes 102
17. The Human Microbiome 105
The Gut Microbiome Kwashiorkor Connection 105
Antibiotics Impact Gut Microbiome and Clostridium difficile Infections 106
18. Bacterial Microfilms 109
Treatment Strategies 110
19. Strategies to Overcome the Problem of Bacterial Antibiotic Resistance 113
Novel Antibacterial Agents 114
Investigations Into Use of Bacteriophage to Fight Bacterial Infections 114
20. Point of Care Diagnostics 117
21. Defining Biomarkers to Help Distinguish Viral Diseases from Bacterial Diseases 119
22. Vaccines in the 21st Century 121
Examples of Active Vaccine Research and Development of Candidate Vaccines in 2016 122
Dengue fever 122
Polio 122
Emerging Problems with use of Attenuated Virus Vaccines 123
Possibilities for Development of Effective HIV Vaccines 124
Vaccine Development 124
Economic Factors in Vaccine Development 125
Vaccines against Ebola 126
23. New Economic Approaches to Financing New Medications 127
References 128
Part V Nuclei, Chromosomes, Genomes and Gene Products 133
24. Entering Genetics through Studies on Nuclei, Chromatin and Chromosomes 135
Training in Tissue Culture and Cytogenetics 136
Progress in Understanding Disorders of Sexual Development 137
Key Genes in Male Sexual Differentiation 139
Key Genes in Female Sexual Development 139
25. Excursions into Enzymes, Polymorphisms and Biochemistry 141
Adventures in Biochemical Genetics 141
Testing Hypotheses on the Subunit Structure of the ADH1, ADH2 and ADH3 Gene Products 142
Molecular Genetic Studies on Alcohol Dehydrogenase 143
Additional ADH Gene Loci 143
Molecular Analyses of Individual Variation of ADH Genes 143
Use of Polymerase Chain Reactions (PCR) to Reveal ADH Polymorphisms 144
Class I ADH Genes and Population Studies 145
Clinical Relevance of ADH Studies 145
Fetal Alcohol Exposure 146
26. Gene Mapping and Somatic Cell Genetics 149
Human Gene Mapping 149
Somatic Cell Hybrids 150
27. Catching up on Developments in Molecular Genetics through the 1960s and 1970s 153
Intense Efforts on Molecular Biology followed Discovery of the DNA Structure 154
Enzymes and the Molecular Revolution 155
Polymerases 155
Reverse Transcriptase 155
Restriction Endonucleases 155
Ligases 156
Southern Blots 156
DNA Sequencing 156
The Impact of the Molecular Cloning Manual of Sambrook, Fritsch and Maniatis 157
Interrupted Genes 157
Incorporation of DNA Technology into Human Gene Mapping and Linkage Studies 158
DNA markers for human gene mapping and linkage studies 158
DNA Amplification through use of the Polymerase Chain Reaction (PCR) 159
Discovery of Polymorphic Markers through Application of PCR Technology 160
Physical Mapping of Markers to Chromosomes 160
In situ hybridization 160
Advances in Nucleic Acid Sequencing Techniques 161
28. The Human Genome Project and Advances in Genetics and Genomics 163
Early Fruits of the HGP: A Map of Sequence Tagged Sites 164
Draft Sequence of the Human Genome 164
Next-Generation Sequencing Methods 165
Massively parallel sequencing 165
Third Generation Sequencing Methods 166
Nanopore Sequencing 166
Analysis of Functional Elements in the Genome 166
Chromatin Architecture and Transcription 167
Non-protein-coding RNA 167
Relevance of Information on the Human Genome Sequence and of Transcription Information for Clinical Medicine 168
Microarrays 168
Analysis of a Specific Gene or Analysis of a Gene Panel 169
Whole Exome or Whole Genome Sequencing 169
Mutation Types and Consequences 170
Genome-wide Association Studies 171
Impact of Gene Interactions 171
Genome-wide Association Studies and Information on Susceptibility to Medication Toxicity 172
DNA Analysis in Forensics 172
DNA Analysis in Cancer 173
Hereditary Cancer Syndromes 174
Analysis of Gene Expression in Tumors 174
Cancer Databases 174
Circulating Tumor DNA and Liquid Biopsy 175
Exosome Shedding by Tumors 175
Emerging Plasma Biomarkers in Cancer Diagnoses 176
29. Gene Regulation and “Endless Forms Most Beautiful” 177
Interconnected: A Gene Shared Family — the Homeodomain Genes 178
Temporal and Spatial Differences in Gene Expression at Different Life Stages 179
Differences in Expression of Products of a Gene 179
Enhancers 179
Promoters 180
Insights into Transcription and its Control 182
One Gene, Different Transcripts, Different Proteins 183
Cell Type Specific Splicing 183
Regulation of Exon Usage 184
Impact of RBFOX1 Gene Expression Levels 184
Regulatory Functions of RNA 185
What Happens to mRNA Spliced out from Introns Contained in Primary RNA Transcripts? 186
Long Non-coding RNAs 186
Epigenetics 186
Nucleotide Modifications 187
Chromatin, Histones: Architecture and Modifications 187
Patterns of Histone Modifications and Gene Expression 188
Chromatin Architecture and Chromatin Remodeling 188
Transcription Factors 189
Nutrition and Epigenetics 189
Environmental Factors and Epigenetics 189
Deficiencies on our Understanding of Gene Regulation 189
References 190
Part VI Genetic Variation and Diseases 199
30. Founder Mutations, Specific Genetic Disorders in Different Countries 201
31. Hypercholesterolemia, Heart Disease and Atherosclerosis 203
Low-density Lipoprotein Receptor (LDLR) Mutations 204
Additional Causes of Raised LDL Cholesterol Levels 205
32. Cardiomyopathy 207
Criteria to Determine that a Mutation is Pathogenic 209
33. Porphyria 211
A Diligent Physician and an Amazing Story 211
Porphyria Variegata 212
Discovery of the Enzyme Defect in Variegate Porphyria and Characterization of Gene Defect 213
Porphyria New York 213
34. Gaucher Disease 217
35. Hemoglobins and Hemoglobinopathies 219
History of Hemoglobin and Hemoglobinopathies 220
Hemoglobin in Patients with Anemia 220
Application of Molecular Biology to the Study of Thalassemias 222
Thalassemias 224
Alpha Thalassemia 225
Alpha Thalassemia and Clinical Manifestations in World Populations 226
Other Hemoglobinopathies that Occur with High Frequencies in Specific Populations 227
Hemoglobin E (HbE) 227
Hemoglobin C (HbC) 228
My Interest in Hemoglobins Takes a Personal Turn 228
My Later Involvement with Hemoglobinopathies 230
Hemoglobinopathies in Europe 231
Current Comprehensive Programs for Treatment of Severe Hemoglobinopathies 231
Sickle cell disease 231
Thalassemias 231
New Approaches to the Treatment of Beta Globin Hemoglobinopathies 232
Investigations into Regulators of Gamma Globin Production 232
Pre-clinical Studies Investigating Gene Editingto Treat Hemoglobinopathies 233
36. Hemophilia 235
Hemophilia: Breakthroughs and Setbacks 236
Treatment of Hemophilia 237
Isolation, Cloning and Characterization of FVIII and FIX Genes 237
Management of Treatment of Hemophilia in Recent Decades 238
Additional Setbacks: Development of Inhibitors to Infused FVIII or FIX 238
Gene Therapy in Hemophilia 239
The Future: Possibilities for Gene Editing in Hemophilia 239
37. Cystic Fibrosis 241
A History of Cystic Fibrosis 241
Transformative Reports 242
Genetics of Cystic Fibrosis 243
The CFTR Gene and its Gene Product 244
CFTR Mutations and their Functional Effects 244
General Treatment in Cystic Fibrosis 246
Molecular Screening for Therapeutic Agents 246
Other Phenotypes that Occur in Patients with CFTR Mutations 247
38. Inborn Errors of Metabolism 249
A First Case 249
Phenylketonuria, Newborn Screening and Inborn Errors of Metabolism Clinic 250
Advances in the Treatment of PKU 251
Advances in Analysis and Treatment of Inborn Errors of Metabolism 251
Clinical Presentations of Inborn Errors of Metabolism 252
Approaches to the Treatment of Inborn Errors of Metabolism 252
Lysosomal Storage Diseases 253
Progress in Enzyme Replacement Therapy 254
Chaperone Therapy 255
References 255
Part VII Complexities and Striving for Insights 265
39. Evolution of Therapies, Complexities, Root Causes, and Moves Toward Personalized Medicine 267
Therapies in Modern Times 267
Addressing Root Causes 267
Complex Causation 269
Genome-wide Association Studies (GWAS) 269
Gene–Environment Interactions and Natural Selection 270
The Move Toward Personalized Precision Medicine 271
40. Psychiatry 273
New Insights through Comprehensive Genetic Studies 273
Demonstration of Polygenic Risk in Psychiatric Disorders 274
41. Aging 277
Genomic Instability 277
Epigenetic Changes in Aging 278
Proteostasis 279
Mitochondrial Function Deterioration and Aging 280
Nutrient Sensing 280
Cellular Senescence 280
Stem Cell Exhaustion 281
Intercellular Communication 281
Brain Plasticity 281
Neurogenesis in the Adult Brain 282
Aging, Neurodegenerative Disease and Advances 283
42. Neurodegenerative Diseases 285
Evidence for Origin of Aggregates in One Region and Spread to Other Regions 286
Clinical Features 286
Family Studies and Genetics 286
Alzheimer’s Disease 287
Parkinson’s Disease 288
Amyotrophic Lateral Sclerosis (ALS) 288
Tau Protein in Neurodegenerative Diseases 289
Therapeutic Approaches 290
References 290
Part VIII The Future 295
43. Stem Cells and Pluripotent Stem Cells 297
Somatic Cell Nuclear Fusion 297
Studies on Teratomas and Teratocarcinomas 298
Embryonic Stem Cells Derived from the Blastocyst 298
Studies Leading up to the 2006 Discovery of Induced Pluripotency in Cells by Takahashi and Yamanaka 298
Vectors used to Transfer Pluripotency Factors 299
Protocols to Establish Induced Pluripotent Stem Cells (IPS cells) 299
Use of IPS Cells as Models to Study Disease-associated Cellular Defects 300
Pluripotent Stem Cells and Cardiomyopathies 301
IPS Cells and Studies of the Aging Process 301
Pluripotent Stem Cells and Regenerative Medicine 302
Cellular Therapies using Bone Marrow-derived Cells 303
Tissue and Organ Chips 303
44. Gene Editing 305
Gene Editing Technologies 305
Clinical Trials of Gene Edited Cells 308
45. Antisense Oligonucleotides 309
46. Towards the Future of Cancer Diagnosis and Therapy 311
Targeted Therapies in Clinical Use for Cancer 312
Liquid Biopsies: Analysis of Circulating Tumor Nucleic Acids 312
Exosomes 312
Cancer Metastases 313
Exosomes 313
Changes in Metabolism in Tumors 313
Immunotherapy in Cancer 314
Immune Modulators 314
Adaptive Cell Transfer and Immunotherapy 315
Tumor Antigens 316
Engineered T Cell Receptors in Cancer Therapy 316
Cancer Vaccines 316
Lipid-complexed Nucleic Acids, Dendritic Cells and Cancer Immunotherapy 317
The Cancer Moonshot 318
Tumor Recurrences 319
Tumor Heterogeneity and Therapy Resistance 320
New Approaches to Early Detection of Tumors 320
New Approaches to Clinical Trials 321
47. Improved Health, Prolonged Survival and Wellness 323
The Wellness Concept 323
48. Health: Human and Planetary 325
Expanding Concepts of Health and of the Dimensions of Environmental Impact 325
Planetary Health 327
ARBO viruses 329
Diseases Caused by ARBO Viruses 330
Zika ARBO virus 330
Convergence 331
References 331
Index 337