title: Gene Therapy Protocols: Volume 1: Production and In Vivo Applications of Gene Transfer Vectors (Methods in Molecular Biology)
author: Joseph LeDoux
description: <P>In Gene Therapy Protocols, Volumes 1 &amp; 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. Todays 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 first volume of Gene Therapy Protocols: Production and In Vivo Applications of Gene Transfer Vectors, readers will find a comprehensive resource of current and emerging methods for the production of viral and non-viral gene transfer vectors, as well as detailed protocols for critical applications in stem cell biology, cancer, diabetes, HIV and tissue engineering. This first 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. </P>
zlib_text: CHAPTER 5
  Gene Targeting
  ERIC KMIEC, PH.D.
  BACKGROUND AND CHALLENGES
  The availability of cloned genes and deoxyribonucleic acid (DNA) sequences, com-
  bined with the ability to transfer and express genes in mammalian cells has revolu-
  tionized biology. Already, therapeutic proteins like tissue plasminogen activator
  (TPA), erythropoietin (EPO), and interferon (IF) have helped thousands of patients
  realize the beneﬁts of molecular medicine. Recent progress in this ﬁeld has raised
  the expectation that genes may be used as therapeutic agents. Such approaches,
  which rely either on puriﬁed proteins or genes, are additive, that is, the defective
  gene (or gene product) is supplemented by the therapeutic drug while the defec-
  tive gene and its products are ignored.
  The “gene addition” approach, however, is plagued by a variety of problems.The
  most damaging of these limitations is the inability to control the expression of the
  newly added gene, due, in part, to the lack of precision in locating t