Gene Editing - InVivo Biosystems

CRISPR/Cas9 off target effects: Hyperactive cutting or media hype?

CRISPR, Gene Editing, View from the Bench / By Ben Jussila / October 17, 2018 November 10, 2020

There has been a lot of media coverage in recent months surrounding CRISPR/Cas9 and the potentially life changing impact the gene-editing technology could have. More recently though, this coverage has focused on the potential for serious consequences in the form of off-target effects. The fear with off-target effects is that CRISPR/Cas9 editing is not as …

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Insertion of two loxP sites to produce a knockout (KO) of an embryonic lethal gene

Gene Editing, View from the Bench / By Trisha Brock / October 3, 2018 November 10, 2020

A client wanted a knockout (KO) of an embryonic lethal gene. We could not make this line using our standard methods. Instead, we inserted two loxP sites. One in the first intron of the gene and the second in the 3’utr. After we confirmed this line by PCR and sequencing, we injected this line with …

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An Abnormal Absence of Antibodies: Filling the Gaps with Gene Editing Tools in Zebrafish

Gene Editing / By Ben Jussila / September 19, 2018 November 18, 2020

The zebrafish is an increasingly popular laboratory organism with many experimental advantages. The animal model has been used by biologists to discover the detailed processes that underlie vertebrate development and disease, and study vertebrate haematopoiesis and immune cell interactions1. The zebrafish embryo is also a popular vertebrate model for neurobiology2. Zebrafish research, however, is limited …

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Genome wide associations and the nematode pharynx: ever the twain shall meet

Gene Editing, View from the Bench / By Shawn Lockery, Ph.D., Co-founder / May 7, 2018 November 10, 2020

Question: What might you get if you cross the feeding organ of a roundworm with a genome wide association study? Answer: A cornucopia of novel genes for synaptic transmission, neuromodulation, energy regulation, peptidergic signaling, learning and memory, and more. This riddle, and its answer, are prompted by the convergence of two exciting trends in research …

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Unlocking the Potential of C. elegans for the Study of Rare Disease

Disease Modeling, Gene Editing / By InVivo Biosystems / December 2, 2017 November 10, 2020

Dr. Andy Golden at NIH discusses his work on identifying regulators and interactors of conserved genes in C. elegans that, when mutated, can cause human disease and opportunities to use C. elegans as a model in clinical and translational research.

Modeling Rare Diseases in Caenorhabditis elegans

Disease Modeling, Gene Editing / By Drs. Ben Nebenfuehr and Andy Golden, NIH / July 24, 2017 November 10, 2020

There are approximately 7,000 rare diseases in humans, ~80% of which are monogenic. A rare disease is defined as affecting less than one in 1,500 people. Combined, these rare diseases affect nearly 1 in 10 Americans (25 to 30 million people), and treatments only exist for around 5% of these diseases. Thanks to the advent of whole genome sequencing, the gene(s) responsible for many rare diseases are known, opening the door for more comprehensive studies.

The cerebellum of a brain affected by Niemann-Pick Type C; blue staining shows the dense pockets of lipid accumulations throughout the brain

Searching for a cure for Niemann-Pick Type C using C. elegans

Disease Modeling, Gene Editing / By Terra Hiebert, Ph.D. and Yoanne Clovis, Ph.D. / April 13, 2017 November 10, 2020

Niemann-Pick Type C is a rare and fatal disorder affecting children. We explore the efficacy of new potential therapeutics with the ScreenChip System.