Physiologically responsive gene therapy.

A W Varley; R S Munford

doi:10.1016/s1357-4310(98)01333-1

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Physiologically responsive gene therapy.

A W Varley·R S Munford

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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.

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Animals Disease Models, Animal Gene Expression Regulation Genetic Therapy Humans Transgenes Journal Article Research Support, U.S. Gov't, P.H.S.Review

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A W Varley, R S Munford. 1998. Physiologically responsive gene therapy.. https://doi.org/10.1016/s1357-4310(98)01333-1

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