In Gene Therapy Protocols, Volumes 1 & 2, internationally recognized investigators describe cutting-edge laboratory techniques for the study of Production and In Vivo Applications of Gene Transfer Vectors (Volume 1) and Design and Characterization of Gene Transfer Vectors (Volume 2). The field of gene therapy has undergone remarkable advances, promising to impact human healthcare significantly in the twenty-first century. Today's technologies can deliver genetic material safely and effectively to cells to slow or halt the progression of disease, and to help repair or regenerate damaged or lost tissues. In this second volume of Gene Therapy Protocols: Design and Characterization of Gene Transfer Vectors, readers will find a comprehensive resource of current and emerging methods for the processing and characterization of viral and non-viral gene transfer vectors, as well as promising approaches to design vectors for efficient, targeted and regulated gene delivery and expression. This second volume of the new and completely revised third edition of Gene Therapy Protocols will prove a necessary tool for graduate students and postdoctoral fellows and invaluable to basic and clinical researchers in both industry and academia.

InhaltsangabeTable of Contents 1 Purification of Retrovirus Particles Using Heparin Affinity Chromatography María de las Mercedes Segura, Amine Kamen and Alain Garnier 2 Scaleable Purification of Adenovirus Vectors John O. Konz, Lee R. Pitts and Sangeetha L. Sagar 3 Quantifying the Titer and Quality of Adenovirus Stocks Fubao Wang, Bill C. Mathis, Allison Montalvo, Jayanthi J. Wolf and John A. Lewis 4 Chromatography-Based Purification of Adeno-Associated Virus Richard H. Smith, Linda Yang and Robert M. Kotin 5 Spectroscopic Methods for the Physical Characterization and Formulation of Nonviral Gene Delivery System Salvador F. Ausar, Sangeeta B. Joshi and C. Russell Middaugh 6 Real Time Multiple Particle Tracking of Gene Nanocarriers in Complex Biological Environments Samuel K. Lai and Justin Hanes 7 Production of Lentiviruses displaying 'Early-Acting' Cytokines for Selective Gene Transfer into Hematopoietic Stem Cells E. Verhoeyen, D. Nègre and F.L. Cossett 8 Fiber-modified Adenoviruses for Targeted Gene Therapy Hongju Wu and David T. Curiel 9 PEGylated Adenovirus for Targeted Gene Therapy Catherine R. O'Riordan and Antonius Song 10 Transposon-Based Mutagenesis Generates Diverse Adeno-Associated Viral Libraries with Novel Gene Delivery Properties James T. Koerber and David V. Schaffer 11 Photochemical Enhancement of DNA Delivery by EGF Receptor Targeted Polyplexes Anette Bonsted, Ernst Wagner, Lina Prasmickaite, Anders Høgset and Kristian Berg 12 Reducing the Genotoxic Potential of Retroviral Vectors Ali Ramezani, Teresa S. Hawley and Robert G. Hawley 13 Evaluation of Promoters for Use in Tissue-Specific GeneDelivery Changyu Zheng and Bruce J. Baum 14 Adenovirus-Mediated Transduction of Auto- and Dual-Regulated Transgene Expression In Mammalian Cells Valeria Gonzalez-Nicolini and Martin Fussenegger 15 Regulated expression of Adenoviral Vectors-based Gene Therapies: Therapeutic Expression of Toxins and Immune-modulators James F. Curtin, Marianela Candolfi, Mariana Puntel, Weidong Xiong, A.K.M. Muhammad, Kurt Kroeger, Sonali Mondkar, Chunyan Liu, Niyati Bondale, Pedro R. Lowenstein and Maria G. Castro 16 Liver-Directed Gene Therapy Using the Sleeping Beauty Transposon System Lalitha R. Belur, R. Scott McIvor and Andrew Wilber 17 Generation and Functional Analysis of Zinc Finger Nucleases Toni Cathomen, David J. Segal, Vincent Brondani and Felix Müller-Lerch 18 Conditional Gene Expression and Knockdown Using Lentivirus Vectors Encoding shRNA Jolanta Szulc