Improving Lives by Modeling Rare Genetic Disorders

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Relief for patients with rare genetic disorders are often out of reach, due to the expense of developing genetically engineered animal models. That's why the Rare Genomics Institute, Taconic Biosciences, and Jonah's Just Begun came together to develop and characterize a model of Sanfilippo Syndrome. Sanfilippo Syndrome is a progressive, devastating rare genetic disorder affecting approximately 1 in 70,000 children. There are four subtypes of the disease,...  Read More

Congenic Mice: What's in a Name?

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Researchers unfamiliar with mouse genetics can be confused by complex strain nomenclature, leaving them unsure how to select controls for a particular test strain. The nomenclature of congenic mice records a wealth of useful information, including both donor and recipient strains and mutations of interest. One common example of this is confusion around background strain for GEM and mutant lines. While it is common now to make...  Read More

Using CRISPR to change "A" to "T" and "C" to "G"

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The use of this acronym has become ubiquitous in the life sciences. For those who don't know, CRISPR stands for Clustered Regularly Interspersed Palindromic Repeats. We hear people talking about it in the context of gene editing of cell lines and animal models. There are now scientific conferences devoted to CRISPR technology, CRISPR in the clinic and CRISPR engineering of animal models. While the intellectual property landscape...  Read More


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