Prenatal Gene Therapy: Concepts, Methods, and Protocols

ISBN-13: 9781493959112 / Angielski / Miękka / 2016 / 392 str.

Charles Coutelle; Simon N. Waddington

Opis

This Methods in Molecular Biology (TM) book covers gene therapy, fetal medicine and medical ethics, with protocols for vector production, test animal breeding and husbandry, surgical gene delivery in large and small animals and methods of gene transfer analysis.

Spis treści

1. The Concept of Prenatal Gene Therapy 

Charles Coutelle, Simon Waddington

2. Candidate Diseases for Prenatal Gene Therapy

Anna L David, Simon Waddington

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

Charles Coutelle, Simon Waddington

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

Raul Alba, Andrew H Baker and Stuart A Nicklin

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

Steven J Howe and Anil Chandrashekran

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

Christopher J Binny, Amit C Nathwani

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

Suet Ping Wong, Orestis Argyros and Richard P Harbottle

8. Use of Manipulated Stem Cells for Prenatal Therapy

Jessica L. Roybal, Pablo Laje, Jesse D. Vrecenak, Alan W. Flake

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

Vedanta Mehta, Donald Peebles, Anna L. David

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

Jessica L. Roybal, Masayuki Endo, Suzanne M.K Buckley, Bronwen R. Herbert, Simon N. Waddington, Alan W. Flake

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

Khalil N. Abi-Nader, Michael Boyd, Alan Flake, Vedanta Mehta, Donald Peebles, Anna L. David

12. Animal Models for Prenatal Gene Therapy:  The Non-human Primate Model

  Citra N Mattar, Arijit Biswas, Mahesh Choolani, Jerry KY Chan

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

Juliette Delhove, Ahad A. Rahim, Tristan R. McKay, Simon N. Waddington, Suzanne M.K. Buckley

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

Vedanta Mehta, Khalil N. Abi-Nader, David Carr, Jacqueline Wallace, Charles Coutelle, Simon Waddington, , Donald Peebles, Anna L. David

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

Charles Coutellle, Simon Waddington, Michael Themis

16. Monitoring for Potential Adverse Effects of Prenatal Gene Therapy:

Genotoxicity Analysis in vitro and on Small Animal Models ex vivo and in vivo

Michael Themis

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

Charles Coutelle , Richard Ashcroft

1. The Concept of Prenatal Gene Therapy 

Charles Coutelle, Simon Waddington

2. Candidate Diseases for Prenatal Gene Therapy

Anna L David, Simon Waddington

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

Charles Coutelle, Simon Waddington

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

Raul Alba, Andrew H Baker and Stuart A Nicklin

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

Steven J Howe and Anil Chandrashekran

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

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

Suet Ping Wong, Orestis Argyros and Richard P Harbottle

8. Use of Manipulated Stem Cells for Prenatal Therapy

Jessica L. Roybal, Pablo Laje, Jesse D. Vrecenak, Alan W. Flake

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

Vedanta Mehta, Donald Peebles, Anna L. David

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

Jessica L. Roybal, Masayuki Endo, Suzanne M.K Buckley, Bronwen R. Herbert, Simon N. Waddington, Alan W. Flake

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

Khalil N. Abi-Nader, Michael Boyd, Alan Flake, Vedanta Mehta, Donald Peebles, Anna L. David

12. Animal Models for Prenatal Gene Therapy:  The Non-human Primate Model

  Citra N Mattar, Arijit Biswas, Mahesh Choolani, Jerry KY Chan

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

Juliette Delhove, Ahad A. Rahim, Tristan R. McKay, Simon N. Waddington, Suzanne M.K. Buckley

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

Vedanta Mehta, Khalil N. Abi-Nader, David Carr, Jacqueline Wallace, Charles Coutelle, Simon Waddington, , Donald Peebles, Anna L. David

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

Charles Coutellle, Simon Waddington, Michael Themis

16. Monitoring for Potential Adverse Effects of Prenatal Gene Therapy:

Genotoxicity Analysis in vitro and on Small Animal Models ex vivo and in vivo

Michael Themis

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

Charles Coutelle , Richard Ashcroft

Blurb

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.