Cellular gene therapy: An overview

H. G. Klein

Research output: Contribution to journalArticle

Abstract

Cellular gene therapy is a technique in which a functioning gene is inserted ex vivo into somatic cells to correct an inborn genetic error or to provide some new function to the cell. The gene-modified cells, often selected and expanded, are infused into the patient. Successful therapy requires gene identification and isolation, efficient insertion and expression, and sufficient circulation or engraftment of the corrected cells for effective therapy. A variety of gene insertion techniques have been employed, but current strategies use a variety of replication-defective virus-like vectors. Most of the clinical studies have involved either lymphocytes or hematopoietic progenitor cells as the gene targets, however numerous other target cells are possible. Certain strategies take advantage of a survival advantage of the gene-corrected cells in the particular patient population, while other strategies involve using gene insertion to protect the desired cell population from the cytotoxic effects of chemotherapy. While few patients have derived definite benefit from the recent cellular gene therapy trials, a great deal of information regarding therapeutic strategy, cell biology, host immune response, and techniques for preparing cellular biologics has derived from these pioneering attempts. Examples of early and ongoing clinical trials will be discussed.

Original languageEnglish (US)
Pages (from-to)243
Number of pages1
JournalInfusionstherapie und Transfusionsmedizin
Volume24
Issue number4
StatePublished - 1997
Externally publishedYes

ASJC Scopus subject areas

  • Hematology
  • Immunology
  • Food Science

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