CRISPR Mouse for Drug Discovery

The expansion of CRISPR from cells to organisms promises a range of benefits, including the production of stably modified animals suitable for drug screening. CRISPR offers many advantages over more traditional genetic engineering methods, including speed and cost reduction (compared to breeding strategies that do not usually work), scalability (compared to transient gene expression, RNAi or most other knock-down methods), and the ability to target or insert any gene that is compatible with the survival of the organism. Possible genetic modifications include knockout, knock-in, knock-down, and gene editing animals.

Unlike specially bred mice that may exhibit only one or two phenotypic or genomic features, CRISPR-edited mouse model models can encapsulate much of the complexity of disease states. The combination of traditional and modern mouse genome editing tools, and the increased genetic diversity in the new modeling system, have synergistic effects that help make mice better models for biomedical research, enhancing the potential for preclinical drug discovery and personalized medicine.

Fig. 1 Pipeline of CRISPR–Cas-assisted drug discovery. (Fellmann C, et al., 2017)

Solution

CRISPR/Cas9 Platform^CB can provide accurate mouse models containing mutations in the same disease genes found in patients, and these models allow for allele-specific treatment outcome assessment. Our CRISPR-edited mouse models can encapsulate the many complexities of disease states such as NAFLD, familial hypercholesterolemia, cardiovascular disease, Alzheimer's disease, and of course cancer.

In addition, we can reveal new drug targets through CRISPR screens and can design mouse models to mimic diseases to improve translatability when searching for new compounds and moving along the pathway of lead optimization and candidate compound delivery. We can also apply this technology to in vivo target validation or generate new targets in vivo to further enhance the preclinical development of disease mouse models and improve clinical relevance. We are committed to providing CRISPR-based gene therapy solutions for a variety of diseases to accelerate drug development and reduce compound attrition rates.

We offer a variety of solutions for the development of drugs based on CRISPR mouse models, including but not limited to

Drug pathway analysis and target identification
Drug target protein structure analysis
Drug screening
Pharmacokinetic assays
Drug activity testing
Drug toxicity testing

Our Advantages

Our models can better simulate the complexity of human disease.
We can provide drug target validation and drug development services.
We can provide an evaluation of therapeutic options in genetically defined systems.
We can reduce compound attrition rates and improve target validation.
We can save time, reduce animal use and overall cost-effectiveness.

Related Services

Related Products

CRISPR/Cas9 Platform^CB is dedicated to providing global biotechnology and pharmaceutical clients with leading drug development services for preclinical stage innovation drug development. Our services cover the full range of our clients' early-stage drug development needs, and we are committed to assisting our clients in completing all phases of preclinical studies for new drug development quickly and efficiently.

References:

Zuberi A, Lutz C. Mouse Models for Drug Discovery. Can New Tools and Technology Improve Translational Power?. ILAR J. 2016, 57(2):178-185.
Fellmann C, et al. Cornerstones of CRISPR-Cas in drug discovery and therapy. Nat Rev Drug Discov. 2017, 16(2):89-100.

For research use only. Not intended for any clinical use.

Inquiry