Gene Therapeutics

Methods and Applications of Direct Gene Transfer

  • Jon A. Wolff

Table of contents

  1. Front Matter
    Pages i-xviii
  2. Background

  3. Methods and Mechanisms

    1. Front Matter
      Pages 81-81
    2. Martin E. Dowty, Jon A. Wolff
      Pages 82-98
    3. Arun Singhal, Leaf Huang
      Pages 118-142
    4. Henry C. Chiou, George L. Spitalny, June R. Merwin, Mark A. Findeis
      Pages 143-156
    5. Patricia L. Chang
      Pages 157-179
    6. Leonard M. Neckers
      Pages 180-192
    7. Ning-Sun Yang, Carolyn De Luna, Liang Cheng
      Pages 193-209
    8. Sergei I. Sukharev, Alexander V. Titomirov, Vadim A. Klenchin
      Pages 210-232
  4. Applications

    1. Front Matter
      Pages 233-233
    2. E. Antonio Chiocca, Julie K. Andersen, Yoshiaki Takamiya, Robert L. Martuza, Xandra O. Breakefield
      Pages 245-262
    3. Kenneth W. Culver, R. Michael Blaese
      Pages 263-280
    4. Joseph C. Glorioso, Neal A. DeLuca, William F. Goins, David J. Fink
      Pages 281-302
    5. Christopher H. Evans, Paul D. Robbins
      Pages 320-343
    6. Leslie D. Stratford-Perricaudet, Michel Perricaudet
      Pages 344-362
    7. Jeffrey M. Leiden, Eliav Barr
      Pages 363-381
    8. Matthew G. Dunckley, George Dickson
      Pages 391-410
  5. Back Matter
    Pages 411-420

About this book


During the first half century of genetics, coinciding with the first half of this cen­ tury, geneticists dreamt of the repair of genetic disease by altering or replacing defective genes. H. J. Muller wrote of the great advantages of mutations, "nanoneedles" in his apt term, for delicately probing physiological and chemical processes. In the same spirit, genes could be used to provide treatments of needle point delicacy. Yet, during this period no realistic possibility appeared; it remained but a dream. The situation changed abruptly at the half century. Microbial genetics and its offshoot, cell culture genetics, provided the route. Pneumococcus transformation showed that exogenous DNA could become a permanent part of the genome; yet attempts to reproduce this in animals produced a few tantalizing hints of success, but mostly failures. Transduction, using a virus as mediator, offered a better op­ portunity. The fITSt reproducible in vivo gene therapy in a whole animal came in 1981. This was in Drosophila, with a transposable element as carrier. Flies were "cured" of a mutant eye color by incorporation of the normal allele, and the effect was transmissible, foreshadowing not only somatic, but germ line gene therapy. At the same time, retroviruses carrying human genes were found to be ex­ tremely efficient in transferring their contents to the chromosomes of cultured cells.


DNA Promoter Translation gene expression gene therapy genes transcription

Editors and affiliations

  • Jon A. Wolff
    • 1
    • 2
    • 3
  1. 1.Department of PediatricsUniversity of Wisconsin Medical SchoolMadisonUSA
  2. 2.Department of Medical GeneticsUniversity of Wisconsin Medical SchoolMadisonUSA
  3. 3.Department of NeurologyUniversity of Wisconsin Medical SchoolMadisonUSA

Bibliographic information