Small Molecule-Triggered Cas9 Protein with Improved Genome-Editing Specificity

By K. Davis, V. Pattanayak, David B. Thompson, John A. Zuris, David R. Liu. 2015

392 citations recorded by the source corpus.

Directly modulating the activity of genome-editing proteins has the potential to increase their specificity by reducing activity following target locus modification. We developed Cas9 nucleases that are activated by the presence of a cell-permeable small molecule by inserting an evolved 4-hydroxytamoxifen (4-HT)-responsive intein at specific positions in Cas9. In human cells, conditionally active Cas9s modify target genomic sites with up to 25-fold higher specificity than wild-type Cas9.

Subfields: Cardiology and Cardiovascular Medicine, Molecular Biology

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In Cardiology and Cardiovascular Medicine

  1. Haifeng Wang
  2. Lei S. Qi
  3. Marie La Russa

In Molecular Biology

  1. Haifeng Wang
  2. Marie La Russa
  3. Lei S. Qi

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