Gene editing technology is expected to treat diseases such as cancer and HIV

The CRISPR/Cas system is an acquired immune system in most bacteria and all archaea, and its full name is the frequent palindrome repeat cluster/every palindrome repeat cluster-associated protein system, which can eliminate foreign matter. A phage or a bacterium that leaves a foreign gene fragment in its genome as a "memory." In recent years, CRISPR/Cas9-based gene editing technology has shown great application prospects in a series of gene therapy applications, such as AIDS, blood diseases, tumors and other human ills; then the recent CRISPR-Cas9 gene editing system What breakthroughs have been made in the treatment of cancer and HIV? Xiaobian has taken stock of this and will study with you!

细看基因编辑技术治疗癌症、HIV等疾病研究进展

[1] Using CRISPR to treat cancer?

According to the National Institutes of Health (NIH), the US Recombinant DNA Advisory Committee (RAC) next week will review the University of Pennsylvania's first clinical trial of the use of revolutionary gene editing technology, CRISPR, to treat human cancer. Using CRISPR technology, scientists can accurately cut target DNA.

This clinical study will extract T cells from the immune system from cancer patients. Next, the researchers will use CRISPR to genetically modify T cells and inject the modified T cells back into the patient so that they will target the destruction of tumor cells.

Carrie Wolinetz, deputy director of science policy at NIH, disclosed the review in a blog post. The cancer immunotherapy being developed at the University of Pennsylvania is aimed at targeting myeloma, melanoma and sarcoma.

The CRISPR technology was developed less than four years ago, but it is already rushing into clinical applications. Prior to this, Editas Medicine, a biotechnology company based in Cambridge, Mass., said it plans to launch a clinical trial in 2017 to treat a rare eye disease using CRISPR.

[2]Cell: Let CRISPR shine in the cancer field

The first CRISPR-Cas9 gene editing technique was used to systematically target every gene in the genome in an overall biological model. From the Broad Institute and the Massachusetts Institute of Technology David H. A team of scientists at the Koch Comprehensive Cancer Institute pioneered the use of this technology to systematically “knock out” (close) all genes in the entire genome of a cancer animal model, revealing genes involved in tumor evolution and metastasis, This paves the way for similar research in other cell types and diseases. The research work was published online in the March 5 issue of Cell.

"The entire senior author, a core member of the Harvard-MIT Broad Institute, a researcher at the MIT McGovern Brain Institute, and an assistant professor at the Massachusetts Institute of Technology in Brain, Cognitive Science and Bioengineering, said: "All The genomic guide RNA (gRNA) library is a powerful screening system, and we began to apply it to animal models to study gene function with excitement. This research is to use Cas9 to identify cancer in vivo and The first step in important genes in other complex diseases."

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