Article

Regulated control of gene therapies with a drug induced switch

2020-02-24

Abstract excerpt

To date, gene therapies for human application rely on engineered promoters that cannot be finely controlled. Here, we report a universal switch element that allows precise control for gene silencing or gene replacement after exposure to a small molecule. Importantly, these small molecule inducers are in human use, are orally bioavailable when given to animals or humans, and can reach both peripheral tissues and th...

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Literature Corpus work: cb0be2f3-a7ce-581d-94d8-96e817b0f602
DOI: 10.1101/2020.02.21.956664

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