About the Book
Gene therapy is expected to revolutionize the practice of medicine at the turn of the third Millennium. Therapeutic/prophylactic benefits should arise from both gene transfer and gene repair/inactivation protocols devised for patients' somatic cells. Gene expression cassettes, designed for the production of therapeutic proteins and non-coding RNA, are thus tested together with emerging gene repair/inactivation techniques on a variety of inherited, acquired and infectious/parasitic diseases, including complex neuro-degenerative processes. This work presents a collection of chapters on the main aspects of gene therapy, some of which have already been treated in the past, and updates and further develops the current survey. It is aimed at understanding why human gene therapy is likely to be a medical breakthrough, although definitive clinical success still needs time to be accomplished. The contributions focus on both technical/biomedical concepts and on experimental/clinical data that appears to confer potential universality to gene therapy. This book does not claim to provide an exhaustive review of the pathologies, which are currently approached with gene therapy.
The presentation of current and emerging approaches, together with problems and tentative issues aims to serve as a booster for the development of new applications in every field of medicine and should, therefore, be of value and interest to researchers in this exciting field.
Table of Contents:
Gene therapy - transient or long-term minigene expression and gene repair/inactivation, R. Bertolotti; Higher-titer retroviral pseudotype vectors for specific targeting of human CD4-positive cells, J. Stitz, P. Muller, H. Merget-Millitzer and K. Cichutek; Lentiviral vectors for gene therapy, A. Follenzi and L. Naldini; Helper dependent adenoviral vectors - improved safety and expression, M.A. Morsy and C.T. Caskey; Adeno-associated virus: basic biology and vector applications, C. Summerford, J. Bartlett and R.J. Samulski; Herpes simplex virus vectors for gene therapy, D.S. Latchman; Liver-selective nucleic acid delivery using the asialoglycoprotein receptor, T. Fukuma, G.Y. Wu and C.H. Wu; Receptor-mediated gene transfer based on plasmid DNA complexes, R. Kircheis, I. Wightman, M. Ogris, S. Brunner, M. Kursa, S. Schuller and E. Wagner; Gene delivery by guanidinium-cholesterol cationic lipids - a 1999 update, N. Oudrhiri, R. Ramasawmy, J.-P. Vigneron, M. Hauchecorne, R. Toury, S. Riquier, E. Vivien, M. Peuchmaur, J. Navarro, J.-M. Lehn and P. Lehn; Progress of fusigenic viral-liposomes, Y. Kaneda, H. Nakashima, N. Tsuboniwa and R. Morishita; Gene therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency, D.B. Kohn; Transgenic rescue of tyrosine hydroxylase-deficient mice - application for generating animal models with cateholamine dysfunction, K. Kobayashi and T. Nagatsu; Adeno-associated virus (AAV) vectors and gene therapy of Parkinson's disease, K. Ozawa, S. Muramatsu, K. Ikeguchi, K. Fujimoto, Y. Shen, D.-S. Fan, H. Mizukami, M. Urabe, Y. Hanazono, A. Kume, M. Ogawa and I. Nakono; Bystander effect in suicide gene therapy of cancers, M. Mesnil and H. Yamasaki; Vaccination strategies for advanced melanoma, L. Sun and D. Schadendorf; Apoptin induces tumour-specific apoptosis - potentials for a novel anti-tumour therapy, M.H.M. Noteborn and A. J. van der Eb; Human DNA immunization with regulatory HIV-1 genes, A. Kjerrstrom, S.A. Calarota and B. Wahren; Alphavirus vectors - from RNA replicons to suicidal DNA vaccines; C. Smerdou and P. Liljestrom; Anti-HIV ribozymes in the inhibition of HIV and AIDS, G. Fanning, L.-Q. Sun and G. Symonds; Alteration of pre-mRNA splicing patterns by modified small nuclear RNAs, L. Gorman, D. Schumperli and R. Kole; Single-base conversion of mammalian genes by an RNA-DNA oligonucleotide, K. Yoon. (Part contents).
