A method to enhance delivery and editing efficiencies of CRISPR-Cas9

Case ID:
C16688

Value Proposition

·        Mechanical tuning of stiffness and viscoelasticity results in higher editing efficiency and requires less transfection reagents compared to traditional plasmid-based CRISPR-Cas9 methods.

·        Enhanced cell-substrate interactions between the cells and gels leads to greater uptake of nanoparticles, quicker onset of gene editing, and overall improved gene editing efficiency.

·        Easily scalable, enabling potential use in cell therapy applications.

·        Tool to optimize drug delivery systems or further study the impact of tissue viscoelasticity on drug delivery.

Unmet Need

·        CRISPR-Cas9 gene editing has become one of the most impactful technologies in scientific research, introducing new hope for numerous genetic conditions once thought to be incurable.

·        Efficiency in gene editing and delivery of Cas9 cargo is necessary to transition to translational applications.

·        There are many barriers to efficient gene editing in CRISPR-Cas9.

·        Currently, many researchers focus on modifying Cas9 or developing new delivery systems, and optimization research is ongoing.

·        However, few researchers have explored manipulation of mechanical cues as a mechanism for improving efficiency.

·        Therefore, there is a strong need to improve gene editing platforms for translational applications.

Technology Description

·        Efficient, scalable methods are needed to translate CRISPR-Cas9 technology for drug delivery applications. 

·        Researchers at Johns Hopkins have developed a method to improve CRISPR-Cas9 delivery and Cas9 editing efficiency by tuning the mechanical properties of the cell culture microenvironment.

·        The method utilizes substrates of varying stiffnesses to regulate cellular mechanotransduction.

Stage of Development

·        Proof of concept studies have been completed. Further validation and mechanistic studies are ongoing.

·        Looking for partners to implement as part of a cell therapy system or use as a research tool for optimizing drug delivery systems and studying cell-substrate interactions.

Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
Method for Controlling CRISPR-Cas9 Genome Editing via Matrix Mechanical Properties PCT: Patent Cooperation Treaty PCT PCT/US2024/030230   5/20/2024     Pending
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For Information, Contact:
Vera Sampels
vsampel2@jhu.edu
410-614-0300
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