Model Generation Solutions INSIGHTS

Unique in its complexity, the central nervous system (CNS) has long represented a challenge for biomedical research, in part because the definition and characterization of constituent cell types remains incomplete. Indeed, one of the major goals behind the 2013 National Institutes of Health (NIH) launch of The Brain Research Through Advancing Innovative Neurotechnologies (BRAIN) Initiative was to "identify and provide experimental access to the different brain cell...  Read More

Site-specific recombinases represent one of the most versatile tools available to scientists engaged in the generation and use of genetically engineered models (GEMs). Chief among these is Cre recombinase, discovered in the P1 bacteriophage and named as such because it causes recombination (Cre) between specific DNA sequences referred to as loxP (locus of crossover (x) in P1) sites, each of which are 34 base pairs in length1....  Read More

Genetically engineered models (GEMs) serve as a backbone of drug discovery. Yet, accessing the most relevant GEM for a particular research objective is not always a simple task. Licensing issues can create hurdles for investigators who need a model ideally suited to their study objectives. Intellectual property (IP) factors must be considered early on; otherwise, a researcher could inadvertently violate a model's licensing restrictions, delaying the study...  Read More

Most viruses have a narrow range of species in which they can establish an infection, for example, a virus that infects humans may not be able to infect laboratory research animals such as mice. This species-specificity presents researchers with a unique problem when studying human viruses. There are two means to address this — one is to modify the virus so that it can infect the target...  Read More

CRISPR/Cas9 is an extremely useful tool for both academic and commercial researchers. It is also widely used in the generation of custom mice and rats from licensed animal model vendors. However, there are multiple patents covering this technology and understanding the differences between them is essential before utilizing CRISPR/Cas9 for model generation. This Insight will focus on two key CRISPR/Cas9 patents and how they have influenced model...  Read More

The application of CRISPR/Cas9 as a mammalian gene editing technology has tremendously matured over the last decade. This has paved the way for the development of CRISPR/Cas9 as therapeutic tool to treat human genetic disease, while making it easier to create the genetically modified animal models required to screen potential therapeutics. In Vivo CRISPR/Cas9-Based Therapeutics in Clinical Trials Current clinical trials utilizing this technology focus on gene...  Read More

The first genetically-modified mouse capable of transferring its modified genome to offspring was developed in the early 1980s. Since then, multiple genetic modification techniques have been developed to modify rodent genomes and are still commonly used today. Random and targeted transgenesis are commonly used today to study the effects of incorporating exogenous DNA for the purposes of expressing proteins not normally present in mice and rats, while...  Read More

In the short time since the introduction of CRISPR technology in 1987, it has proved to be a valuable tool. In 2018, there were over 17,000 publications on CRISPR gene editing technology compared to seven years earlier when there were less than 100 publications. The future is bright in how CRISPR will impact research, treatments, and cures. VOX recently published, A simple guide to CRISPR, one of...  Read More

It was recently announced that the legal battle over the CRISPR/Cas9 patents is likely finished with the Broad Institute prevailing above UC Berkeley. This ongoing debate began in 2014 with multiple research groups and institutions claiming rights to the CRISPR/Cas9 technology. The researchers involved in this case are Dr. Jennifer Doudna (UC Berkeley) and Dr. Emmanuelle Charpentier (University of Vienna) who engineered the Cas9 endonuclease from a...  Read More

CRISPR/Cas9 genome editing technology enables the modification of the mouse and rat genomes with unprecedented simplicity and speed but is subject to limitations that restrict its application to the introduction of simple mutations such as constitutive knockout or point mutation alleles. Easi-CRISPR overcomes those limitations by using longer single-stranded DNA, offering researchers a faster and more efficient technology for targeted transgenesis and complex genetic modifications. Limitations of...  Read More