Physiologically responsive gene therapy

Alan W. Varley, Robert S. Munford

Research output: Contribution to journalReview articlepeer-review

30 Scopus citations

Abstract

The goal of physiologically responsive gene therapy is to allow a host's endogenous regulatory mechanisms to control the production of therapeutic proteins (effectors). Ideally, effector production would be switched on in response to specific signals, stay within therapeutic limits and be switched off when no longer needed. In this way, the unwanted consequences of constitutive, high-level effector expression could be avoided. While recent studies have shown that transgenes can be regulated within animal hosts, they have also highlighted significant problems that require much further research.

Original languageEnglish (US)
Pages (from-to)445-451
Number of pages7
JournalMolecular Medicine Today
Volume4
Issue number10
DOIs
StatePublished - Oct 1 1998

ASJC Scopus subject areas

  • Molecular Medicine
  • Genetics

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