Preface xi
Acknowledgments xiii
About the Companion Website xv
1 Introduction 1
1.1 The Basics of Molecular Medicine 2
1.2 The Human Cell 4
1.3 DNA Replication and Gene Expression 10
1.4 Biological Communication 26
1.5 The Immune System 31
2 Methods in Molecular Medicine 41
2.1 DNA Microarrays 42
2.2 Quantitative Polymerase Chain Reaction 45
2.3 Next-generation Sequencing 51
2.4 Proteomics 58
2.5 Animal Models in Biomedical Research 65
2.6 Alternatives to Animal Testing 68
2.7 Additional Methods 74
3 Genetic Disorders 81
3.1 Single-gene Disorders 82
3.2 Polygenic Disorders 101
4 Molecular Oncology 109
4.1 Basics of Oncology 110
4.2 Selected Cancer Diseases 112
4.3 Oncolytic Virus Therapy 127
4.4 Cancer Stem Cells 128
4.5 Cancer Immunotherapy 130
5 Molecular Virology 139
5.1 The Basics of Virology 140
5.2 Vaccination 149
5.3 Detection of Viruses 159
5.4 Antiviral Therapy 163
5.5 Prions 172
6 Bacteria and Eukaryotic Pathogens 177
6.1 Bacteria 178
6.2 Eukaryotic Pathogens 192
7 Genomics 199
7.1 Whole Genome Sequencing 200
7.2 The Human Genome 202
7.3 Sequencing of Ancient DNA 209
7.4 Follow-up Initiatives of the Human Genome Project 211
8 Genetic Testing 219
8.1 Types of Genetic Tests 220
8.2 Chromosome Abnormalities 224
8.3 Molecular Diagnosis 229
9 Pharmacogenetics/Pharmacogenomics 239
9.1 Uptake and Transport of Drugs 242
9.2 Drug Metabolism 242
9.3 Drug Targeting 247
9.4 Drug Toxicity and Hypersensitivity 250
9.5 Drug Development and Individual Pharmacotherapy 250
10 Recombinant Protein Drugs 255
10.1 Production of Recombinant Proteins 258
10.2 Classes of Recombinant Drugs 264
11 Gene Therapy 287
11.1 Types of Gene Therapy 288
11.2 Methods of Gene Transfer 289
11.3 Tissue Specificity of Gene Transfer and Gene Expression 297
11.4 Applications of Gene Therapy 300
11.5 Genome Editing with CRISPR/Cas 305
11.6 Future Prospects 309
12 Stem Cells 313
12.1 Embryonic Stem Cells 315
12.2 Adult Stem Cells 318
12.3 Induced Pluripotent Stem Cells 320
12.4 Transdifferentiation and Direct Reprogramming 323
12.5 Differentiation of Stem Cells 325
12.6 Medical Applications of Stem Cells 328
13 Oligonucleotide-based Strategies 341
13.1 Antisense Oligonucleotides (ASOs) and Ribozymes 343
13.2 RNA Interference 351
13.3 MicroRNAs 361
13.4 Aptamers, Decoy Oligonucleotides, and Immunostimulatory Oligonucleotides 367
14 Ethics in Molecular Medicine 383
14.1 The Basis of Bioethics 384
14.2 Fields of Application 386
Abbreviations 399
Glossary 405
Index 409
