Prenatal Gene Therapy

Chapter 1 The Concept of Prenatal Gene Therapy

Chapter 2 Candidate Diseases for Prenatal Gene Therapy

Chapter 3 Vector Systems for Prenatal Gene Therapy: Choosing Vectors for Different Applications

Chapter 4 Vector Systems for Prenatal Gene Therapy: Principles of Adenovirus Design and Production

Chapter 5 Vector Systems for Prenatal Gene Therapy: Principles of Retrovirus Vector Design and Production

Chapter 6 Vector Systems for Prenatal Gene Therapy: Principles of Adeno-Associated Virus Vector Design and Production

Chapter 7 Vector Systems for Prenatal Gene Therapy: Principles of Non-viral Vector Design and Production

Chapter 8 Use of Manipulated Stem Cells for Prenatal Therapy

Chapter 9 Animal Models for Prenatal Gene Therapy: Choosing the Right Model

Chapter 10 Animal Models for Prenatal Gene Therapy: Rodent Models for Prenatal Gene Therapy

Chapter 11 Animal Models for Prenatal Gene Therapy: The Sheep Model

Chapter 12 Animal Models for Prenatal Gene Therapy: The Nonhuman Primate Model

Chapter 13 Choice of Surrogate and Physiological Markers for Prenatal Gene Therapy

Chapter 14 Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Use of Large Animal Models with Relevance to Human Application

Chapter 15 Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Mouse Models for Developmental Aberrations and Inadvertent Germ Line Transmission

Chapter 16 Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Genotoxicity Analysis In Vitro and on Small Animal Models Ex Vivo and In Vivo

Chapter 17 Risks, Benefits and Ethical, Legal, and Societal Considerations for Translation of Prenatal Gene Therapy to Human Application