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The Development of Human Gene Therapy

(Cold Spring Harbor Monograph Series 36)

Book Series:  Cold Spring Harbor Monograph Series
Subject Area(s):  Gene TherapyHuman Biology and DiseaseMolecular Biology

Edited by Theodore Friedmann, University of California, San Diego

© 1999 • 729 pp., illus., color plates, index
Cloth • $140 28.00
ISBN  978-087969528-6
You save: 80%

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The idea of human gene therapy was accepted by the medical community and society at large long before believable clinical benefits began to emerge. In this book, some of the field's most distinguished contributors chronicle the evolution of this momentous direction for medicine, illustrating how imaginative concepts shaped the development of technologies and brought the daring new idea to its current position of imminent practical success. This is a book designed to endure as clinical advances accumulate, a clear-eyed work of reference that will anchor the further development of this revolution in therapy. It is an essential addition to libraries of clinical medicine, biotechnology, and public policy, and a resource that no laboratory investigator with an interest in the biology of gene transfer should be without.


Chapter 1: The Origins, Evolution, and Directions of Human Gene Therapy (T. Friedmann)
Chapter 2: Retroviral Vectors (J.-K. Yee)
Chapter 3: Lentiviral Vectors (L. Naldini and I.M. Verma)
Chapter 4: Structure and Genetic Organization of Adenovirus Vectors (M.M Hitt, R.J. Parks, and F.L. Graham)
Chapter 5: Adenovirus Vectors (N.A. Wivel, G.-P. Gao, and J.M. Wilson)
Chapter 6: Strategies to Adapt Adenoviral Vectors for Gene Therapy Applications: Targeting and Integration (P.N. Reynolds and D.T. Curiel)
Chapter 7: Adeno-associated Viral Vectors (R.J. Samulski, M. Sally, and N. Muzyczka)
Chapter 8: Gene-transfer Tool: Herpes Simplex Virus Vectors (S. Laquerre, W.F. Goins, S. Moriuchi, T.J. Oligino, D.M. Krisky, P. Marconi, M.K Soares, J.B. Cohen, J.C. Glorioso, and D.J. Fink)
Chapter 9: Emerging Viral Vectors (D.J. Jolly)
Chapter 10: Advances in Synthetic Gene-delivery System Technology (P.L. Felgner, O. Zelphati, and X. Liang)
Chapter 11: Receptor-mediated Gene Delivery Strategies (M. Cotten and E. Wagner)
Chapter 12: Naked DNA Gene Transfer in Mammalian Cells (J.A. Wolff)
Chapter 13: Delivery Systems and Adjuvants for DNA Vaccines (J.B. Ulmer and M.A. Liu)
Chapter 14: Targets for Gene Therapy (T. Friedmann and A.G. Motulsky)
Chapter 15: The Hematopoietic System as a Target for Gene Therapy (B.P. Sorrentino and A.W. Nienhuis)
Chapter 16: Cancer Immunotherapy (D. Pardoll and G.J. Nabel)
Chapter 17: Gene Transfer and Stem-cell Transplantation (M.K. Brenner)
Chapter 18: Suicide Genes: Gene Therapy Applications Using Enzyme/Prodrug Strategies (J.C. Morris, R. Touraine, O. Wildner, and R.M. Blaese)
Chapter 19: The Logic of Anti-angiogenic Gene Therapy (J. Folkman, P. Hahnfeldt, and L. Hlatky)
Chapter 20: Apoptosis as a Goal of Cancer Gene Therapy (J.C. Reed)
Chapter 21: Advances in Gene Therapy for HIV and Other Viral Infections (E.M. Poeschla and F. Wong-Staal)
Chapter 22: Progress Toward Gene Therapy for Nervous System Diseases (A. Martínez-Serrano and A. Björklund)
Chapter 23: Targeted Gene Repair in Mammalian Cells Using Chimeric RNA/DNA Oligonucleotides (E.B. Kmiec, B.T. Kren, and C.J. Steer)
Chapter 24: Human Gene Therapy: Public Policy and Regulatory Issues (N.A. Wivel and W.F. Anderson)
Chapter 25: Ethical Issues in Human Gene Transfer Research (E.T. Juengst and L. Walters)


review:  "Highlights of this large volume include an excellent chapter on naked DNA injection into various tissues. The fact that this leads to gene expression at all is remarkably encouraging for the future. Another chapter considers targeted gene repair in mammalian cells using chimaeric oligonucleotides. Although a long way from the clinic, this represents the beginnings of genetic surgery. The short review on stem-cell transplantation is concise, yet informative, while the ethics of the subject are well reviewed in a historical context."

review:  "The Development of Human Gene Therapy is a benchmark for the field in 1999. The book should be of interest to scientists and nonscientists alike. Friedmann has captured the prevailing feeling that efforts must be focused on the basic principles of gene transfer to translate the science into effective therapeutics in the future."
      —Nature Medicine

review:  "The Development of Human Gene Therapy is a timely and useful book that highlights the significant recent advances in the field. At the same time, it illuminates the important hurdles that remain to be overcome en route to a clinically useful reality."

review:  "Publication of The Development of Human Gene Therapy comes at an opportune time, given the upheavals that the gene therapy field has experienced over the past few years. The book is edited by the highly experienced Ted Friedmann, who not only was one of the first advocates of gene therapy but also has become an astute chronicler of the field's recent ups and downs…. Friedmann has succeeded in putting together a meticulous and timely book that admirably captures the gene therapy roller-coaster ride over the past decade."
      —Molecular Therapy