An important new collection of clinical and preclinical reports on genetic therapy, this book describes illustrative examples of diseases in which gene-based interventions are presently plausible, and presents case studies of current research using both synthetic oligonucleotides and biological vectors.
Combining the insights of over 50 contributors, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors

  • furnishes a historical overview of genetic therapy
  • highlights official Food and Drug Administration positions on the preparation of oligonucleotides and vectors
  • offers practical models of agent preparation, animal testing, pharmacokinetics, toxicology, and clinical trials
  • discusses both synthetic DNA and biological vector approaches to cancer, viral, and cardiological indications
  • illustrates for new practitioners how each stage of genetic therapy is developed
  • details genetic treatment of leukemia; lymphoma; cancer of the brain, breast, colon, kidney, and lung; melanoma; HIV; and coronary restenosis
  • includes examples of antisense, ribozyme, tumor suppressor, immunostimulation, and gene replacement therapy
  • and addresses questions of preparation, delivery, toxicity, mechanism, and specificity.