GEMs Design INSIGHTS


CRISPR Patent Update: Broad vs. Berkeley

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

Application of CRISPR/Cas to the Generation of Genetically Engineered Mice White Paper

UPDATE: This White Paper has been updated in 2018 to reflect the latest technological advances and usage recommendations of CRISPR/Cas in the generation of research models. CRISPR/Cas gene editing has emerged as a versatile technology for inducing precise genetic alterations in a number of different species, including mice and rats. Given the importance of genetically engineered mice in modeling human disease, the power of CRISPR has the...

Read More

The Advantages and Limitations of Easi-CRISPR

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

Deflating the CRISPR/Cas9 Off-Target Controversy

UPDATE: The paper which originally tied CRISPR/Cas 9 to off-target mutations in the mouse genome has been retracted. More information is available here. The use of CRISPR/Cas9 has profoundly affected mouse genetics by reducing the cost and the timeline associated with the introduction of mutations in the genome. But is it possible that CRISPR isn't specific enough for the generation of genetically engineered animal models? Since its...

Read More

Webinar Q&A — Advanced Toolkit for Syngeneic Tumor Modeling in Mice

Did you miss Dr. Philip Dubé's recent webinar, Advanced Toolkit for Syngeneic Tumor Modeling in Mice? His presentation introduced advanced concepts and critical considerations for studying immuno-oncology mechanisms and therapeutics in mice. Webinar Synopsis: Syngeneic models are becoming more advanced due to the availability of genetically engineered mouse models. This advanced toolkit makes it possible to study the many intricate mechanisms through which the immune system controls...

Read More

Webinar Q&A — Your Guide to the C57BL/6 Mouse

Did you miss Dr. Philip Dubé's recent webinar, The Most Important Mouse in the World - Your Guide to the C57BL/6 Mouse? His presentation explored genetic diversity amongst C57BL/6 substrains, the implications for GEM models, and the utility of the C57BL/6 mouse in studies of metabolism, immunology, oncology and behavior. Due to time constraints, many of the questions researchers submitted to his Q&A session went unanswered. Here...

Read More

Here is what CRISPR, the number 62 and pigs have in common!

In December of 2015, Taconic Biosciences published an Insight titled, What do CRISPR, the number 62, and pigs have in common? In that Insight, Dr. George Church of Harvard Medical School explained that his lab had edited 62 genes in pig embryos using CRISPR-Cas9. Their goal was to make pigs better candidates as organ donors for humans. Given their close anatomical size to humans, pigs are thought...

Read More

Genetic Variation in C57BL/6 ES Cells and Knockout Mice

Genetically engineered animal models (GEMs) are powerful tools for biomedical research and drug discovery, but genetic variation in animal models derived from distinct ES cell lines and subsequent breeding can impact both the interpretation and reproducibility of studies which employ them1. While recent advances in gene editing accelerated the migration of models to a pure C57BL/6 background, researchers should remain aware of potentially confounding results from genetic...

Read More

Cystic Fibrosis Treatment from Vertex May Restore Lung Function

Cystic Fibrosis (CF) is a genetic disorder that affects the lungs, digestive system, and a host of metabolic functions. In humans, CF is a progressive disease, where chronic dysfunction of muco-ciliary clearance leads to severe congestion, thick mucus build-up, and severe persistent lung infections. In 1960 the median life expectancy for a patient with CF was ten years old. However, with significant advances in research and clinical...

Read More

Oh Transgene, Where Art Thou?
Mapping Transgene Insertion

Over the last three decades, transgenic mice have become a critical in vivo modeling tool in biomedical research. Transgenic technique1, whereby an exogenous gene is inserted into the mouse genome by direct injection of DNA into the pronuclei of a zygote, has enabled thousands of new transgenic lines to be created2. However the technique is not without limitations. One of the biggest drawbacks of using pronuclear injection...

Read More