Prenatal Gene Therapy

The Concept of Prenatal Gene Therapy.-Candidate Diseases for Prenatal Gene Therapy.-Vector Systems for Prenatal Gene Therapy: Choosing Vectors for Different Application.-Vector Systems for Prenatal Gene Therapy: Principles of Adenovirus Design and Production.-Vector Systems for Prenatal Gene Therapy: Principles of Retrovirus Vector Design and Production.-Vector Systems for Prenatal Gene Therapy: Principles of Adeno-Associated Virus Vector Design and Production.-Vector Systems for Prenatal Gene Therapy: Principles of Non-viral Vector Design and Production.-Use of Manipulated Stem Cells for Prenatal Therapy.-Animal Models for Prenatal Gene Therapy: Choosing the Right Model.-Animal Models for Prenatal Gene Therapy: Rodent Models for Prenatal Gene Therapy.-Animal Models for Prenatal Gene Therapy: the Sheep Model.-Animal Models for Prenatal Gene Therapy: The Non-human Primate Model.-Choice of Surrogate and Physiological Markers for Prenatal Gene Therapy.-Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Use of LargeAnimal Models with Relevance to Human Application.-Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Mouse Models for Developmental Aberrations and Inadvertent Germ Line Transmission.-Monitoring for Potential Adverse Effects of Prenatal Gene Therapy: Genotoxicity Analysis in vitro and on Small Animal Models ex vivo and in vivo.-Risks, Benefits and Ethical, Legal and Societal Considerations for Translation of Prenatal Gene Therapy to Human Application.