Transgenic mice harboring CRISPR-based transcriptional activators

CRISPR-based tools for gene regulation have enabled new studies of gene function and the development of genetic therapies. However, these studies have largely been limited to experiments with cells in culture due to the limitations of delivery of these tools in vivo. To facilitate in vivo targeted gene activation with CRISPR technology, we have created transgenic mice that conditionally express the dCas9-p300 activator under the control of Cre recombinase. Delivery of a gRNA and Cre in vivo, either by gene delivery or genetic cross breeding, leads to targeted gene activation in select tissues or cell types.

Duke File (IDF) Number

T-006938

Inventor(s)

  • Gersbach, Charles
  • Gemberling, Matthew

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College

Pratt School of Engineering