Physiologically responsive gene therapy
AW Varley, RS Munford - Molecular medicine today, 1998 - cell.com
AW Varley, RS Munford
Molecular medicine today, 1998•cell.comThe 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 …
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 …
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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