Molecular Therapeutics: 21st Century Medicine

1 Introduction
1.1 Microbial diseases
1.2 Cancer and heart disease
1.3 Genetic diseases
1.4 Role of molecular biology in therapeutics

2 Prenatal diagnosis and pre-implementation

2.1 Should we treat inherited diseases?
2.2 Genetic screening
2.2.1 Pre-implementation genetic diagnosis
2.3 Counselling

3 Simple protein replacement therapy
3.1 Preventing transfusion-transmissible infectious
diseases in the UK
3.2 Ensuring the safety of organ transplants
3.3 Preventing transfusion-transmissible infectious
diseases worldwide
3.4 HIV

4 Recombinant protein production
4.1 Choice of organism
4.2 Alternatives to E. coli for the production of
recombinant proteins
4.3 Problems with recombinant protein production
4.4 All recombinants must be tested before they are
given to humans
4.5 Why make recombinant proteins?
4.6 Recombinant products
4.7 Generics

5 Recombinant vaccines
5.1 Vaccine history
5.2 Vaccines
5.3 Vaccine methods
5.4 Types of vaccine
5.5 The limitations of vaccine programmes
5.6 The role of the WHO
5.7 Problems specific to developing countries
5.8 Economics and logistics of vaccinology
5.9 Recombinant vaccines
5.10 Rational design: bioinformatics and proteomics
5.11 Other interesting areas for vaccine development
5.12 Conclusion

6 Therapeutic antibodies and immunotherapy
6.1 Monoclonal antibodies
6.2 Monoclonal production
6.3 Therapeutic monoclonal antibodies
6.4 Transgenic monoclonals
6.5 The uses of monoclonal antibodies in therapy
6.6 Specific examples of therapeutic strategies
6.7 Other recombinant proteins used in immunotherapy

7 Transgenic animals
7.1 Why do we want to engineer the genomes of animals?
7.2 Experimental procedure
7.3 DNA constructs, insertional mutagenesis and
homologous recombination
7.4 Uses of inducible and tissue-specific promoters
7.5 Introduction of the DNA into the cells
7.6 Uses of transgenics

8 Transplantation: a form of gene therapy
8.1 Introduction
8.2 Bone marrow
8.3 Solid organ transplantation
8.4 Other cells and tissues
8.5 Summary of the problems associated
with transplantation
8.6 Transplantation statistics
8.7 Legislation
8.8 Religious beliefs and transplantation

9 Xenotransplantation
9.1 Introduction
9.2 Rationale for the use of non-human donors
9.3 Organs from non-human primates
9.4 Pigs
9.5 Problems with pigs
9.6 Government legislation
9.7 When will xenotransplantation start?
9.8 Patient attitudes
9.9 Ethics
9.10 Alternatives to xenotransplants

10 Reproductive cloning
10.1 History
10.2 Problems
10.3 Why was there so much interest in Dolly?
10.4 Was Dolly a lone example?
10.5 Why is cloning useful?
10.6 Is human cloning a reality?
10.7 Why can we not produce human clones that
are identical?
10.8 So why clone humans?
10.9 What are the ethical and moral problems?

11 Stem cell therapy
11.1 The potency of cells
11.2 Cloning
11.3 Potency of stem cells
11.4 Potential sources of stem cells
11.5 Stem cells and therapeutic cloning
11.6 Legislation and therapeutic cloning
11.7 Other sources of stem cells
11.8 What can be done?
11.9 Experiments on embryonic cells
11.10 Experiments on fetal tissue and cord blood
11.11 Stem cells from adult tissues
11.12 Safety and technical problems
11.13 Perceived scope of therapy
11.14 Clinical trials of stem cell therapy
11.15 What are the future prospects for stem cell research?

12 Gene augmentation therapy
12.1 Introduction
12.2 Strategy

13 Gene therapy trials for inherited diseases
13.1 Introduction
13.2 Examples of disease treated with retroviral gene therapy

13.3 Cystic fibrosis
13.4 Animal trials with Factor IX
13.5 Adenoviruses have also been used to introduce
genes into brain
13.6 Duchenne's muscular dystrophy
13.7 Problems with adenoviruses
13.8 The uses of adeno-associated viruses
13.9 Liposome vector trials
13.10 Trials with polymer mareix delivery

14 Gene silencing technologies
14.1 Antisense therapy
14.2 Triple helix (triplex) technology
14.3 Ribozymes
14.4 Small interfering RNAs (siRNAs)

15 Gene therapy for cancer
15.1 What causes cancer?
15.2 Cancer: a multifactorial disease
15.3 Cancer statistics
15.4 Best treatment currently available
15.5 Do chemo- and radiotherapy cause problems?
15.6 New cancer therapies
15.7 Cancer models in animals
15.8 What kinds of gene therapy can we use to
treat cancer?
15.9 Perceived problems in cancer gene
augmentation therapy
15.10 Gene silencing technologies and cancer
15.11 Conclusion

16 Single-nucleotide polymorphisms (SNPs)
and therapy

17 Legislation, clinical trials and ethical issues
17.1 Legislative bodies
17.2 Clinical trials
17.3 The problems of placebo controlled trials
17.4 The need for informed consent
17.5 Trials in developing countries
17.6 Recent trial issues
17.7 Conclusion

Epilogue
Sourcing references

Index