CRISPR Chinese cattle are more than Zhang Feng, this scholar has repeatedly published many articles to improve CRISPR

Release date: 2017-02-16

As a co-founder of Nasdaq's "first" Editas company, David R. Liu, a professor of chemical and chemical biology at Harvard University and a researcher at the Howard Hughes Medical Institute, is also a prominent CRISPR researcher in his research group. In a recent paper published in Nature Biotechnology, researchers increased the scope of CRISPR gene targeting and reduced the edit box from 5 nucleotides to 1-2 nucleotides, adding base editing. Accuracy.

In the case of CRISPR research, David R. Liu has been working to improve this key technology for genome editing. In 2013, he joined Professor Jennifer Doudna of the University of California at Berkeley, taking the lead in a detailed analysis of Cas9, revealing Cas9. The precision of the DNA sequence that can be directed to its programming, and the sensitivity of this protein/RNA complex to the bait off-target sequence.

Since then, the results have been continuous, such as the article published in Nature last year, realized that CRISPR only "cut" a single base breakthrough. Most of the diseases associated with genetic variation are point mutations, and existing genetic methods are not very effective at repairing point mutations. His proposed method provides more evidence for the ability of CRISPR to cleave only a single base.

At the end of last year, the research team also published a review in Cell magazine, which summarizes the CRISPR-based technologies that enable mammalian genome editing, and the various applications of these technologies, highlighting gene editing techniques in basic research, biotechnology, and disease treatment. Some significant progress in other areas.

A recent paper, published in the journal Nature Biotechnology, points out that the Cas9 base editing method from Streptococcus pyogenes has many advantages, such as the need to form double-stranded DNA nicks, or donor DNA templates. This group of researchers reported the use of genetic engineering and natural Cas9 mutations in different PAMs to achieve five C to T (or G to A) basic editing, increasing the range of gene targeting. Moreover, reducing the edit box from 5 nucleotides to 1-2 nucleotides increases the accuracy of base editing.

This study further improved the CRISPR technique, enabling differentiation of adjacent C nucleotides, preventing similar editing, and doubling the number of disease-related targets Cs. This laid the foundation for the clinical application of CRISPR technology.

David R. Liu has also been working on the clinical application of CRISPR. He has said that “it is well known that the ability to manipulate our genomic structure may have a profound impact on human health. But when you give patients some will change them. Before the treatment of the gene, you need to be very certain that it will not cause unexpected effects elsewhere, because cutting a target site as well as off-target sites may mean causing treatment of the disease or triggering cancer to form two different endings. ."

“An important piece of information is to weigh the balance between activity and specificity. This is an important lesson because when scientists develop these tools into promising treatments, they need to make sure that when they increase activity, they don’t import new ones. Off-target cutting effect."

Original search

Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions

Source: Biopass

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