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Disease Modeling

A View From the Bench: Roadblocks and Frustrations When Expectation Meets Reality.

In an effort to create better models for epilepsy, we’ve been working on a grant-funded project to humanize important synaptic machinery by replacing endogenous coding sequences with human coding sequences.  Syntaxin is a key synaptic protein, encoded by the gene STX1A, that helps synaptic vesicles dock, fuse with the membrane, and release neurotransmitters.  Syntaxin has …

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Zebrafish and C. elegans in ALS: tightening the bench-to-clinic gap

While advances in molecular imaging, high-throughput screening, genomics and techniques like CRISPR-Cas9 gene editing have dramatically enhanced our understanding of the human body and disease, the pace of advancing new treatments for those diseases has lagged behind significantly. One way to shorten the time between laboratory discoveries and available new drugs is to use animal …

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CRISPR technologies enable humanized animal models to aid disease research

C. elegans as a model to evaluate the function of disease genes In 1998, the soil nematode Caenorhabditis elegans became the first multicellular organism of which the genome has been sequenced completely [1]. One surprising result of this approach was that ~65% of the human disease genes have a counterpart in the worm [2]. In …

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Reviewers’ Choice Abstract Award at ASHG Goes to Ben Jusilla for Demonstrating how Zebrafish Can be Used for Disease Modeling

Ben Jusilla, one of our in-house zebrafish experts, presented his poster “Zebrafish modeling for the clinic: Rapid in vivo functional testing of patient variants for clinical applications” at ASHG and won the Reviewers’ Choice Award in the category of Precision Medicine, Pharmacogenomics, and Genetic Therapies. His poster discusses how we use zebrafish as an in-vivo model to measure …

Reviewers’ Choice Abstract Award at ASHG Goes to Ben Jusilla for Demonstrating how Zebrafish Can be Used for Disease Modeling Read More »

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