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Two professors from Arizona State University are among the first beneficiaries of the Somatic Cell Genome Editing (SCGE) grant from the National Institutes of Health Joint Fund. The $ 2,600,000 grant, spread over five years, will fund the first study of CRISPR technology, a genome editing technology, to be used on a "human-on-a-chip liver" platform.
The SCGE program, launched in January 2018, aims to improve therapeutic options for rare and common diseases, including supporting methods to improve the editing of the human genome.
Samira Kiani and Mo Ebrahimkhani, assistant professors at the School of Biological Systems and Health Systems Engineering, combine their expertise in short and regularly spaced palindromic repetitions, or CRISPR, of microphysiological technologies and systems humans to evaluate the safety and effectiveness of genome editing. on the function of human tissues.
CRISPR allows researchers to target genes and genetic material in cells to regulate their behavior and function. Because of its ease of engineering and programmability, CRISPR is considered a breakthrough technology that can potentially help heal diseases, repair damaged body tissues and restore people's health.
However, as with any new technology, applying the CRISPR method can potentially produce unexpected results.
"[CRISPR] is a pathogenic source; So, to talk about the human being, you have to face a number of problems, such as toxicity, an immune response or some other adverse effects that can affect human cell tissues, "said Kiani, Chief It is possible that the introduction of the system creates some sort of non-targeted effects in the genome, which means that it not only influences the target's DNA code, but also unintended changes to the target's DNA code. parts of the genome we do not wear. "t know [about] and do not want to. "
Kiani and Ebrahimkhani will apply the CRISPR method on the Liverchip® platform to identify biomarkers of the human liver genome indicating toxicity. The DNA analysis will also reveal biomarkers indicating the non-targeted effects of Cas9 – the DNA separation enzyme used in CRISPR, which allows for highly accurate editing and regulation of Genoa.
Until now, CRISPR has only been tested on animal models or human cell lines. The use of the Liverchip® platform provides a model that tightly summarizes human biology and will significantly reduce the number of discrepancies introduced by animal models.
These organ-on-chip media are essentially a 3D cell culture system designed to exhibit the specific characteristics of a human body. Several cells in the middle self-assemble to generate a tissue similar to a human organ, mimicking even the blood flow of the human body and the profusion of media in the cells.
"The ultimate goal is to create a culture system that can predict the liver tissue response in humans," said Ebrahimkhani, who worked with this platform while studying at the Massachusetts Institute of Technology. . "In the long run, we hope to be able to develop a candidate CRISPR system that can target a specific gene in humans by controlling cell type, time of action and any potential toxicity. "
The liver will probably be one of the first human organs where gene therapies will be tested. Given the frequency of degenerative genetic diseases associated with metabolism and liver function, the use of the liver-on-chip platform as a substitute for human liver cells is ideal for studying the efficacy of CRISPR / Cas9 as a therapeutic tool.
The multidisciplinary team of investigators includes Jin Park, an assistant research professor at the Virginia G. Piper Center for Personalized Diagnostics at ASU's Biodesign Institute. Park will help analyze data from RNA and DNA sequences to identify biomarkers in tissues. Linda Griffith, professor of innovation teaching in biological and mechanical engineering at the Massachusetts Institute of Technology of the School of Engineering and expert in microphysical systems, and David Hughes of CN Bio Innovations – the commercial supplier of the Liverchip® ASU team in this search. They will bring new technologies and their relevant expertise to human-based cell platforms.
"We are pleased to see that NIH has entrusted Samira and Mo with the leadership of this multi-institutional grant," said Marco Santello, Director of the School of Biological and Health Systems Engineering, one of the six schools of Ira A Fulton Schools of Engineering. "It's a true testament to the caliber of the faculty in Fulton Schools."
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