© 2007 • 793 pp., illus., appendix, index
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Understanding gene function and regulation requires rigorous testing in live cells and organisms. Recent advances have provided a variety of new strategies for delivering DNA and RNA into cells and probing their expression, as well as new clinical applications that rely upon the introduction of genetic material. The vast number of available techniques for clinical and laboratory research often makes selecting the optimal method a difficult process. Gene Transfer: Delivery and Expression of DNA and RNA provides the first comprehensive guide to technical approaches for delivering nucleic acids into cells and organisms and of ensuring (even manipulating) appropriate expression. The detailed, step-by-step protocols cover a variety of methods, both well established and newly evolving. These include viral and nonviral methods of gene delivery, transgenic approaches, strategies for the regulation of transgene expression, and modification of the host response. The introductory matter to each chapter includes concise technical and theoretical discussions with considerations for selection of the appropriate system and strategies for delivery.

  “This volume is a very comprehensive and practical guide describing all the virally, chemically and physically mediated gene transfer techniques I have come across and a few more!...It is a very useful resource.”
      —Society for General Microbiology Quarterly

1. Introduction

T. Friedmann and J. Rossi

VIRAL VECTORS

2. Retroviral Vectors

K. Cornetta, K.E. Pollok, and A.D. Miller

3. Development of Lentiviral Vectors Expressing siRNA

G. Tiscornia, O. Singer, and I.M. Verma

4. HIV–2 Vectors in Human Gene Therapy: Design, Construction, and Therapeutic Potential

K.V. Morris and F. Wong–Staal

5. SIV Vectors as Vehicles for DNA Delivery

E. Verhoeyen, F.–L. Cosset, and D. Nègre

6. Production and Use of Feline Immunodeficiency Virus–based Lentiviral Vectors

D.T. Saenz, R. Barraza, N. Loewen, W. Teo, and E.M. Poeschla

7. Lentivirus Transduction of Hematopoietic Cells

M.–J. Li and J.J. Rossi

8. Spleen Necrosis Virus–based Vectors

Z. Parveen, M. Mukhtar, and R.J. Pomerantz

9. Foamy Virus Vector Production and Transduction of Hematopoietic Cells

N.C. Josephson and D.W. Russell

10. Simian Foamy Virus Type–1 Vectors

J. Park and A. Mergia

11. Generation of VSV–G–pseudotyped Retroviral Vectors

J.–K. Yee

12. Targeted Gene Transfer with Surface–engineered Lentiviral Vectors

E. Verhoeyen and F.–L. Cosset

13. Preparation of Pseudotyped Lentiviral Vectors Resistant to Inactivation by Serum Complement

G.H. Guibinga and T. Friedmann

14. Generation of 2A Peptide–linked Multicistronic Vectors

A.L. Szymczak–Workman, K.M. Vignali, and D.A.A. Vignali

15. Construction of First–generation Adenoviral Vectors

P. J. Ross and R.J. Parks

16. Production and Characterization of Helper–dependent Adenoviral Vectors

D.J. Palmer and P. Ng

17. Cell and Tissue Targeting

Y. Kawakami and D.T. Curiel

18. Stable Producer Cell Lines for AAV Assembly

G. Chadeuf and A. Salvetti

19. Strategies for the Design of Hybrid Adeno–associated Virus Vectors

A. Asokan and R.J. Samulski

20. Recombinant Herpes Simplex Virus Vectors

W.F. Goins, D. M. Krisky, J.B. Wechuck, D. Wolfe, S. Huang, and J.C. Glorioso

21. Herpes Simplex Virus Type–1–derived Amplicon Vectors

W.J. Bowers and H.J. Federoff

22. γ–2 Herpesvirus Saimiri–based Vectors

A. Whitehouse

23. Gene Delivery Using HSV/AAV Hybrid Amplicon Vectors

O. Saydam, D.L. Glauser, and C. Fraefel

24. Polyomaviruses: SV40

D.S. Strayer, C. Mitchell, D.A. Maier, and C.N. Nichols

25. SV40 In Vitro Packaging: A Pseudovirion Gene Delivery System

C. Kimchi–Sarfaty and M.M. Gottesman

26. Baculovirus–based Display and Gene Delivery Systems

A.R. Mäkelä, W. Ernst, R. Grabherr, and C. Oker–Blom

27. Safe, Simple, and High–capacity Gene Delivery into Insect and Vertebrate Cells by Recombinant Baculoviruses

K.J. Airenne, O.H. Laitinen, A. J. Mähönen, and S. Ylä–Herttuala

28. Alphaviruses: Semliki Forest Virus and Sindbis Virus as Gene Delivery Vectors

K. Lundstrom

29. Gene Transfer into Mammalian Cells Using Targeted Filamentous Bacteriophage

A. Baird

30. Selection, Isolation, and Identification of Targeting Peptides for Ligand–directed Gene Delivery

M. Trepel, W. Arap, and R. Pasqualini

31. Rescue and Propagation of Tropism–modified Measles Viruses

T. Nakamura and S.J. Russell

32. Picornavirus–based Expression Vectors

S. Mueller and E. Wimmer

33. Reverse Genetics of Influenza Viruses

G.A. Marsh and P. Palese

NONVIRAL TECHNIQUES AND VECTORS

34. An Overview of Condensing and Noncondensing Polymeric Systems for Gene Delivery

D.B. Shenoy and M.M. Amiji

35. Transfection of Hippocampal Neurons with Plasmid DNA Using Calcium Phosphate Coprecipitation

B. Goetze and M. Kiebler

36. Gene Delivery to Skin Using Biolistics

W.C. Heiser

37. Optimizing Electrotransfection of Mammalian Cells In Vitro

S. Li

38. Micro In Utero Electroporation for Efficient Gene Targeting in Mouse Embryos

T. Shimogori

39. Lipoplex and LPD Nanoparticles for In Vivo Gene Delivery

S.–D. Li, S. Li, and L. Huang

40. Bioresponsive Targeted Charge Neutral Lipid Vesicles for Systemic Gene Delivery

W. Li and F.C. Szoka, Jr.

41. HVJ Liposomes and HVJ Envelope Vectors

Y. Kaneda

42. Polylysine Copolymers for Gene Delivery

S.W. Kim

43. PEI Nanoparticles for Targeted Gene Delivery

F. Alexis, J. Zeng, and S. Wang

44. Cyclodextrin–containing Polycations for Nucleic Acid Delivery

J.D. Heidel

45. Bionanocapsules Using the Hepatitis B Virus Envelope L Protein

T. Yamada, J. Jung, M. Seno, A. Kondo, M. Ueda, K. Tanizawa, and S. Kuroda

46. Formulations of Solid Lipid Nanoparticles for Transfection of Mammalian Cells In Vitro

C. Rudolph and J. Rosenecker

47. PEGylated Poly–l–lysine DNA Nanoparticles

P.B. Davis and T.H. Kowalczyk

48. Water–soluble Lipopolymers and Lipopeptides for Nucleic Acid Delivery

R.I. Mahato, Z. Ye, and S.W. Kim

49. Cationic Polysaccharides for DNA Delivery

I. Yudovin–Farber, H. Eliyahu, and A.J. Domb

50. Sustained Release of Plasmid DNAs Encoding Platelet–derived Growth Factor and Hyaluronan Synthase 2 from Cross–linked Hyaluronan Matrices and Films

W. Chen

51. Linear Polyethylenimine: Synthesis and Transfection Procedures for In Vitro and In Vivo

M. Ogris and E. Wagner

52. Protein Nanospheres for Gene Delivery: Preparation and In Vitro Transfection Studies with Gelatin Nanoparticles

S. Kommareddy and M.M. Amiji

53. Vesicular Stomatitis Virus–G Conjugate

A. Miyanohara

54. High–throughput Methods for Screening Polymeric Transfection Reagents

G.T. Zugates, D.G. Anderson, and R. Langer

55. Poly(Lactic Acid) and Poly(Ethylene Oxide) Nanoparticles as Carriers for Gene Delivery

N. S. Csaba, A. Sánchez, and M. J. Alonso

56. Biodegradable Nanoparticles

J.K. Vasir and V. Labhasetwar

57. Transposon–mediated Delivery of Small Interfering RNA: Sleeping Beauty Transposon

B.S. Fletcher

58. Efficient DNA Delivery into Mammalian Cells by Displaying the TAT Transduction Domain on Bacteriophage λ

J. Wadia, A. Eguchi, and S.F. Dowdy

59. Cell–penetrating Peptide–mediated Delivery of Peptide Nucleic Acid Oligomers

P. Lundberg, K. Kilk, and Ü. Langel

REGULATION OF TRANSGENE EXPRESSION

60. Conditional Mutagenesis of the Genome Using Site–specific DNA Recombination

K. Ohtsubo and J.D. Marth

61. Expression and Validation of Ribozyme and Short Hairpin RNA in Mammalian Cells

M. Amarzguioui

62. Mifepristone–inducible Gene Regulatory System

K. Schillinger, X. Ye, S. Tsai, and B.W. O'Malley

63. Dimerizer–mediated Regulation of Gene Expression

V.M. Rivera, L. Berk, and T. Clackson

64. RheoSwitch System: A Highly Sensitive Ecdysone Receptor–based Gene Regulation System Induced by Synthetic Small–molecule Ligands

P. Kumar and A. Katakam

65. Site–specific Integration with Phage φC31 Integrase

R.T. Hillman and M.P. Calos

66. Creating Zinc Finger Nucleases to Manipulate the Genome in a Site–specific Manner Using a Modular–assembly Approach

M. Porteus

SPECIALIZED TECHNIQUES OF GENE AND VECTOR DELIVERY

67. Assembly of De Novo Bacterial Artificial Chromosome–based Human Artificial Chromosomes

J. Basu and H.F. Willard

68. Delivery of Naked DNA Using Hydrodynamic Injection Techniques

D.L. Lewis, M. Noble, J. Hegge, and J. Wolff

69. Nonviral Gene Transfer across the Blood–brain Barrier with Trojan Horse Liposomes

W. M. Pardridge

70. Sonoporation: An Efficient Technique for the Introduction of Genes into Chick Embryos

S. Ohta, O. Yukiko, K. Suzuki, M. Kamimura, K. Tachibana, and G. Yamada

71. Genetic Manipulation of Mammalian Cells by Microinjection

D.W. Rose

72. Magnetofection

C. Plank and J. Rosenecker

73. Photochemical Internalization for Light–directed Gene Delivery

A. Bonsted, A. Høgset, E. Wagner, and K. Berg

TRANSGENIC APPROACHES

74. Pronuclear Microinjection in Mice

W. Tsark

75. Knockdown Transgenic Mice Generated by Silencing Lentiviral Vectors

O. Singer, G. Tiscornia, and I.M. Verma

APPENDIX

Cautions

Subject Index