Gene Transfer Vectors for Clinical Application

Preface: The successful clinical use of viral vectors for human gene therapy.

Theodore Friedmann

1. General principles of retrovirus vector design

Tammy Chang And Jiing-Kuan Yee

2. Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID)

Alain Fischer, Salima Hacein-Bey and Marina Cavazzana-Calvo

3. Retrovirus and lentivirus vector design and methods of cell conditioning

Samantha Cooray, Stephen J Howe and Adrian J Thrasher

4. Analysis of the clonal repertoire of gene corrected cells in gene therapy

Anna Paruzynski, Hanno Glimm, Manfred Schmidt and Christof von Kalle

5. Developing novel lentiviral vectors into clinical products

Anna Leath and Kenneth Cornetta

6. Lentivirus vectors in beta-thalassemia

Emmanuel Payen, Charlotte Colomb, Olivier Negre, Yves Beuzard, Kathleen Hehir and Philippe Leboulch

7. Gene Therapy for Chronic Granulomatous Disease

Elizabeth M. Kang, and Harry L. Malech

8. Alternative splicing caused by lentiviral integration in the human genome

Arianna Moiani and Fulvio Mavilio

9. Genotoxicity assay for gene therapy vectors in tumor prone Cdkn2a^-/- mice

Eugenio Montini and Daniela Cesana

10. Lentiviral Hematopoietic Cell Gene Therapy for X-linked Adrenoleukodystrophy

Nathalie Cartier, Salima Hacein-Bey-Abina , Cynthia C. Bartholomae, Pierre Bougnères , Manfred Schmidt, Christof Von Kalle, Alain Fischer, Marina Cavazzana-Calvo and Patrick Aubourg

11. Retroviral replicating vectors in cancer

Christopher R. Logg , Joan M. Robbins , Douglas J. Jolly , Harry E. Gruber , and Noriyuki Kasahara

12. Adeno-associated virus vectorology, manufacturing and clinical applications

Joshua C. Grieger and R. Jude Samulski

13. Gene Delivery To The Retina: From Mouse To Man

Jean Bennett, Daniel C. Chung, and Albert Maguire

14. Generation of hairpin-based RNAi vectors for biological and therapeutic application

Ryan L. Boudreau and Beverly L. Davidson

15. Recombinant adeno-associated viral vector reference standards

Philippe Moullier and Richard O. Snyder

16. NIH oversight of human gene transfer research involving retroviral, lentiviral and adeno-associated virus vectors and the role of the NIH recombinant DNA advisory committee draft

Marina O’Reilly, Allan Shipp, Eugene Rosenthal, Robert Jambou, Tom Shih, Maureen Montgomery, Linda Gargiulo, Amy Patterson and Jacqueline Corrigan-Curay

17. Regulatory structures for gene therapy medicinal products in the European Union

Bettina Klug, Patrick Celis, Melanie Carr and Jens Reinhardt