This technology utilizes anti-CRISPR proteins for efficient spatial and temporal control of gene editing.
CRISPR-Cas9 is a powerful gene-editing tool that can accurately target and modify specific DNA sequences, potentially correcting disease-causing mutations. However, off-target effects can occur when Cas9 mistakenly cuts DNA at unintended locations, leading to potential harm or unintended consequences. Research indicates that Cas9 activity beyond the first cell cycle leads to mosaicism, wherein cells within an individual have genetic variation. As such, temporal control strategies are needed to minimize the duration of Cas9 activity and, in turn, improve the safety and efficacy of CRISPR-Cas9 gene editing.
This technology proposes the use of anti-CRISPR proteins to restrict Cas9 activity to the first cell cycle, for improved spatial and temporal control of gene editing. By blocking or slowing down the activity of Cas9, anti-CRISPR proteins control gene editing and regulate CRISPR activity to increase gene-editing efficiency and reduce off-target gene editing in an embryo. This technology improves the success of gene editing in the human embryo for CRISPR-based gene therapies.
Patent Pending(US20230295667)
IR CU21215
Licensing Contact: Kristin Neuman
