GEMs DESIGN INSIGHTS


Targeting APOE4 for Alzheimer's Disease Therapies

Past research efforts, focused on Alzheimer's disease (AD) pathology and the genetics of familial AD, led to the discovery of important AD-related proteins, including amyloid-beta (Aβ) and tau. However, efforts to develop AD therapies based on targeting Aβ and tau have largely failed. Years of disappointing trials have brought increasing demand for new AD targets and alternative treatment approaches. Using mice expressing apolipoprotein E4 (apoE4), a human...

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Using HLA Transgenic Mice to Investigate Novel T Cell Therapies

Recent clinical successes in immuno-oncology, coupled with developments in genetically engineered mouse models, are driving interest in utilizing new aspects of immune cell biology for drug discovery. One promising class of cell-based treatments is Chimeric Antigen Receptor T (CAR-T) cell therapies, which take advantage of fundamental mechanisms of immune cell biology to develop novel, tumor-killing T cell therapies. T cell receptors (TCRs) are the antigen recognition machinery...

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Mother's Diet Influences Alzheimer's Disease Outcomes

Increasingly obesity and its associated comorbidities (cardiovascular disease, type 2 diabetes mellitus, hypertension, etc.) are identified as risk factors for Alzheimer's disease (AD). Determining the link between diet and Alzheimer's disease has been challenging, in large part due to obesity's association with numerous systemic physiological changes. Recent studies suggest that poor maternal diet can influence epigenetic changes which predispose offspring to the development of metabolic syndromes. Since...

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Improving Lives by Modeling Rare Genetic Disorders

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,...

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Modeling Proprioception

NPR recently published "How A 'Sixth Sense' Helps Simone Biles Fly, And The Rest Of Us Walk", that explored proprioception - the sense of the relative position of neighboring parts of the body - with a story on children with a rare mutation in a gene called PIEZO2. When Damiana was younger, her condition baffled doctors, says her mother, Diana Sawyer. "She was very late at doing...

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Biotechnology Patents and Genetically Modified Organisms

How do biotechnology patents affect the generation, use and distribution of genetically modified organisms, such as knockout mice or transgenic animal models? What is a licensing agreement and what are the implications of licensing (or not licensing) methods and technologies used in biomedical research? Can you use the new model you recently obtained from a collaborator for your research program? In a nutshell, patents protect intellectual property...

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Unraveling Rare Disease: Taconic's Integrated Solution Supports Vici Syndrome Research

Rare diseases often have a genetic component, so the ability to model a genetic modification and study it in vivo is vital to understanding the disease's mechanisms and identifying therapies. As a leader in the custom development of genetically modified models, Taconic Biosciences is committed to applying our unique capabilities to advance rare disease research. We recently demonstrated that commitment by awarding a $50,000 package of integrated...

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