Primary and Stem Cells: Gene Transfer Technologies and Applications

UMA LAKSHMIPATHY is a principal investigator at Life Technologies. She has a PhD in life sciences, with academic and industry experience in molecular biology and stem cells. Dr. Lakshmipathy holds four patents and has authored more than forty publications. BHASKAR THYAGARAJAN is a program manager at Life Technologies. He has a PhD in pharmacology, with expertise in the areas of molecular biology, DNA recombination, gene and cell therapy, and protein purification. He holds one patent and has authored more than twenty publications.

FOREWORD vii Michele Calos PREFACE ix CONTRIBUTORS xi PART I: CLONING AND GENE DELIVERY 1 1. DNA Assembly Technologies Based on Homologous Recombination 3 Billyana Tsvetanova, Lansha Peng, Xiquan Liang, Ke Li, Jian-Ping Yang, Tony Ho, Josh Shirley, Liewei Xu, Jason Potter, Wieslaw Kudlicki, Todd Peterson, and Federico Katzen 2. Multigene Assembly for Construction of Synthetic Operons: Creation and Delivery of an Optimized All-IN-One Expression Construct for Generating Mouse iPS Cells 19 Takefumi Sone, Yoko Takata, Hiroe Kishine, Taichi Andoh, Fumio Imamoto, and Jonathan D. Chesnut 3. Strategies for the Delivery of Naked DNA 37 Jennifer C. Moore and Ronald P. Hart PART II: NONINTEGRATING TECHNOLOGIES 49 4. Episomal Vectors 51 Junying Yu 5. Nonintegrating DNA Virus 85 Chris Kemp and David Kuninger 6. Nonintegrating RNA Viruses 103 Noemi Fusaki 7. Protein Delivery 119 Xiao-Jian Han and Kazuhito Tomizawa PART III: INTEGRATING TECHNOLOGIES 135 8. Sleeping Beauty Transposon-Mediated Stable Gene Delivery 137 Andrew Wilber, Xianzheng Zhou, Xin Huang, Dan S. Kaufman, and R. Scott McIvor 9. Integrating Viral Vectors for Gene Modifications 163 Gemma L. Mendel, Brian Paszkiet, and Rafal P. Witek 10. Bacteriophage Integrases for Site-Specific Integration 199 Bhaskar Thyagarajan 11. Improving Gene Targeting Efficiency in Human Pluripotent Stem Cells 211 Sangyoon Han, Anna McCann, Louise C. Laurent, Jeanne F. Loring, and Ying Liu PART IV: APPLICATIONS 227 12. Modified Stem Cells as Disease Models and in Toxicology Screening 229 Catharina EllerstrEUROom, Peter Sartipy, Raimund Strehl, and Johan Hyllner 13. Screening and Drug Discovery 251 Elizabeth A. Davenport and Robert S. Ames INDEX 273