Gene editing tools: Building a path to GMP
Using Cas9 in protein form provides benefits that include reduced off-target effects, immediate activity (no need to transcribe and/or translate), and controlled dosage. Aldevron gene editing nucleases are available in defined formulations and custom options, designed for multiple delivery methods, including electroporation, transfection, microinjection and whole animal applications.
A simple solution from R&D to Clinical Material
To increase the simplicity of sourcing material for our clients, Aldevron offers a one-vendor strategy for both pre-clinical and GMP gene editing nucleases. We provide research grade Cas9 nucleases for use in development work, and also a standard GMP product to edit cells targeted for clinical studies. Below is the design of the construct Aldevron has selected for GMP production, which will be available Q4 2017.
How Aldevron's Cas9 Enzymes work
By incorporating nuclear localization signals on one or both termini, Cas9 traverses the nuclear membrane to edit DNA with high efficiency. The following table shows SpCas9 and AsCpf1 nucleases available from Aldevron. We can also use our manufacturing and QC expertise to produce client-specific products.
About Aldevron Cas9
Testimonials for Aldevron Cas 9
"The Aldevron reagent is of the highest quality and is utilized exclusively and extensively for our gene targeting experiments. The high concentration allows us to modify primary cells efficiently and also lets us use guide RNA transcripts that are generated with standard RNA bases. This is an important determinant as the need for specialized chemistries to protect the transcript requires extra time and money."
- Mark Osborn, Ph.D. Minnesota Stem Cell Institute, University of Minnesota
- Read Mark Osborn's blog post on the benefits of high-concentration Cas9
"We saw high knockout efficiency of the engineered Cas9 varieties from Aldevron, compared with competitors we tested. The presence of the nuclear localization signal (NLS) increased the efficiency of gene editing, presumably by helping translocation of the Cas9 ribonuclearprotein to the nucleus. The high concentration of Aldevron’s formulation allows us to use the required amounts of protein without diluting it extensively with other reagents or cell media. Due to these reasons and the repeatability of results we obtain, we have been using Aldevron products for our research involving human gene editing requiring Cas9 protein."