Drug Discovery via CRISPR/Cas System    

The emergence of CRISPR/Cas9 technology offers virtually unlimited opportunities for genetic editing. Not only can it be used as a therapeutic tool, but genome editing tools can also revolutionize drug discovery. In a comprehensive review in 2017, scientists at the University of California at Berkeley, including the co-discoverers of CRISPR/Cas, Jennifer Doudna, concluded that this type of genetic editing "is ready for the direct impact on real-world drug discovery and development.

➢ Screening for target sites

The process of drug discovery begins with the identification of drug targets (genes, RNA transcripts or proteins) associated with the disease of interest. The ability of CRISPR/Cas to help identify target molecules will have a crucial impact on drug discovery. The CRISPR library can detect living cells with specific conditions, such as drug therapy, positive selection can easily elucidate the resistance mechanism, and negative selection can effectively detect dead or slow-growing cells. By using the system, researchers can identify genes and proteins that cause or prevent disease, thereby identifying potential drug targets. Once the putative targets are determined, further functional information is collected by in vitro and in vivo studies. CRISPR can aid in these processes by promoting gene knockout or protein overexpression in cell lines.

➢ Drug discovery

Successful drug development requires appropriate models to aid early drug development decisions, but generate new disease models is a difficult and expensive process in the past. CRISPR/Cas9 has good versatility and has been used in more animal model development. In addition, CRISPR/Cas9 allows multiple genes to be edited at once, which applies to most human diseases that are not single genes. Therefore, CRISPR/Cas can make it easier to create cells that accurately mimic disease and the entire animal model system, which allows scientists to more accurately discovery new drugs and verify the safety and efficacy of the drugs, ensuring that these models better predict what will happen in clinical trials.

The researchers also use CRISPR/Cas to integrate with the cellular DNA repair process - or "knock in" the selected DNA fragment. This can introduce mutations that transform the protein encoded by the target gene to produce a beneficial effect, which can then be designed to be easier to induce. In addition, upregulating or downregulating gene activity using the CRISPR/Cas system is a more subtle way of studying the importance of genes and proteins that can be activated or inhibited by drugs to treat disease.

Figure 1: Pipeline of CRISPR–Cas-assisted drug discovery (Christof Fellmann. 2017)

CRISPR/Cas9 Platform^CB is a leading genetic editing biotechnology company with extensive experience in editing CRISPR genes and the ability to handle a variety of issues. We are committed to solving technical application problems and providing the most professional and comprehensive genetic editing technology solutions for our clients working on the editing of CRISPR/Cas9 genes. To support your projects, we offer custom CRISPR/Cas9 services, including the design and synthesis of gRNAs for specific loci, custom human disease models (animal models and cell line models), and we also provide high-quality products, including CRISPR/Cas9 related kits, Cas9 enzyme. If you have any questions and want to know more about our CRISPR-related services and products, please feel free to contact us.

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