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Promise and Pitfalls: An Updated View of CRISPR/Cas9 for Genetic Engineering in Mice and Rats

 

CRISPR/Cas9 gene editing has revolutionized the generation of genetically engineered mouse and rat models — providing options for both speeding and simplifying the path to production of many different types of mutations.

In many cases, CRISPR/Cas9 gene editing is a relatively simple and cost-effective alternative to gene targeting in embryonic stem (ES) cells. For this reason, it has quickly become one of the leading technologies used for model development. And with the ability to install precise genetic mutations, it is used to produce a wide range of models valuable for both basic and translational research.

This white paper explores how CRISPR/Cas9 gene editing works, its advantages and disadvantages, and the process Taconic employs for custom model generation projects.

Read this white paper to:

  • Understand CRISPR/Cas9 gene editing mechanisms, including how single-guide RNAs are engineered to direct Cas9 to specific target sequences
  • Learn why CRISPR/Cas9 gene editing is more straightforward than gene targeting in ES cells, for suitable projects
  • Discover the advantages of CRISPR/Cas9, including the ability to generate a custom model faster, at a lower cost, and using a wider variety of genetic backgrounds
  • Understand its limitations, such as restrictions on the size and complexity for knock-in projects, off-target mutation risks, and the potential for secondary on-target mutations
  • Find out how Taconic approaches CRISPR/Cas9 model generation projects to ensure a successful outcome

Get Access Here

Did You Know?

Using CRISPR/Cas9 to edit single-cell zygotes can eliminate the need to manipulate ES cells, or to generate a complex targeting construct, saving time and money

CRISPR/Cas9 can be used in cultured cells, such as ES cells, which can benefit projects that require knock-in of a sequence intermediate in size or complexity

CRISPR/cas9 is a versatile gene editing technology that also provides greater flexibility in terms of the genetic backgrounds that may be used

Patrick Gordon, PhD

Scientific Program Manager, Custom Model Generation Solutions

Patrick has 13+ years of experience with genetically engineered mouse models. Apart from his own training and research, this has included the design and oversight of large-scale colony management programs, as well as the design, generation, and molecular validation of new models. He also has deep expertise in the use of Cre/loxP systems for conditional gene expression. Broadly trained in neuroscience, genetics, and developmental biology, Patrick obtained his PhD in neuroscience from the University of Utah.

Christopher Raymond, PhD

Scientific Program Manager, CUstom Model Generation Solutions

Through a 20+ year career spanning the pharmaceutical industry, academia, and now a leading mouse and rat model provider, Chris has applied his background in genetics, genomics, molecular biology, and developmental biology to diverse roles encompassing genetic engineering, drug development, toxicology, and biopharmaceutical research. He has deep expertise in developing translational models to further scientific advances. Chris obtained his PhD in Biochemistry, Molecular Biology, and Biophysics from the University of Minnesota.

Kenn Albrecht, PhD

Associate Director, Custom Model Generation Solutions

For 25+ years Kenn has been engaged in developing and characterizing mouse models of human disease using his training in classical and molecular genetics, genomics, and developmental biology. While at Boston University School of Medicine, where he is now an adjunct faculty member, he led the Transgenic and Genome Engineering core facility, was one of the founding directors of the Genome Science Institute, and ran an NIH-funded research lab. Kenn obtained his PhD in Genetics from the University of Connecticut.

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