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The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions. In Prenatal Gene Therapy: Concepts, Methods and Protocols, expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These include detailed protocols for vector production, for breeding and husbandry of the animal models, for the surgical procedures of gene delivery in large and small animals and for the methods of gene transfer analysis. Written in the highly successful Methods in Molecular Biology(TM) series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls.
Thorough and intuitive, Prenatal Gene Therapy: Concepts, Methods and Protocols seeks to aid scientists in the further study of prenatal disease and gene therapy projects beyond the scope of fetal medicine.
List of contents
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.
Summary
The emerging field of prenatal gene therapy is founded on scientific and technical advances in fetal medicine, molecular biology and gene therapy. This preclinical research subject aims at applying gene therapy during pregnancy for the prevention of human diseases caused by early onset congenital or gestation related conditions. In Prenatal Gene Therapy: Concepts, Methods and Protocols, expert researchers in the field detail many of the protocols which are now commonly used to study gene therapy, fetal medicine and medical ethics. These include detailed protocols for vector production, for breeding and husbandry of the animal models, for the surgical procedures of gene delivery in large and small animals and for the methods of gene transfer analysis. Written in the highly successful Methods in Molecular Biology™ series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and key tips on troubleshooting and avoiding known pitfalls.
Thorough and intuitive, Prenatal Gene Therapy: Concepts, Methods and Protocols seeks to aid scientists in the further study of prenatal disease and gene therapy projects beyond the scope of fetal medicine.
