Cathy Miller

Recent research advances on the CRISPR-Cas9 gene editing tool

Blog Post created by Cathy Miller on Dec 28, 2018

We all know that gene editing technology can help to treat a variety of diseases effectively. However, due to its probability of off-target, this technology also has certain drawbacks. So what research achievements have scientists made in the field of gene editing recently? Let’s take a look.

[1] Nat Med: Is the human body really immune to the CRISPR-Cas9 gene editing tool?

The CRISPR-Cas9 gene editing system has produced exciting results in the field of gene therapy, which has inspired scientists to use the tool to treat human hereditary diseases. Recently, in a research report published in the international journal Nature Medicine, scientists from Germany have studied the immune response of the human body to CRISPR-Cas9 and found that the human body can produce a wide range of immunity against Cas9 protein. Now researchers are developing a variety of innovative strategies to ensure CRISPR-Cas9 gene editing systems can be used safely in a range of clinical applications.

In this study, the researchers analyzed the potential benefits and potential risks of CRISPR-Cas9. CRISPR-Cas9 is a new molecular genetic editing tool that helps scientists make specific changes to the DNA of human organisms, animals and plant organisms. This technology is similar to a special molecular scissors that can help scientists cut, silence or remove specific DNA sequences. The tool can be used to modify cells in vivo or in vitro, or directly transported to the body to directly modify specific targeted cells. Now researchers have already tested in animal models the validity and usability of this gene editing tool.

[2]Science: Successfully restored dystrophin expression in Duchenne muscular dystrophy dog model using CRISPR gene editing technology

Duchenne Muscular Dystrophy (DMD) is one of the most common fatal genetic diseases in children. The incidence of DMD in boys is 1 in 5000. It causes muscle and heart failure and leads to premature death in the early 30s. When the patient's muscles degenerate, they are forced to sit in a wheelchair, and when their diaphragm is weakened, they eventually rely on the respirator to breathe. Although scientists have known for decades that mutations in the dystrophin-encoding gene cause this protein to fail, there is no effective treatment available to date.

In a new study, researchers from the University of Texas Southwestern Medical Center, Exonics Therapeutics, and the Royal Veterinary College of the United Kingdom were the first to use D CRISPR gene editing technology to prevent DMD progression in DMD patients carrying four of the most common mutations. They recorded unprecedented improvements in muscle fibers in these DMD dog models.

[3] Nature: Editing of human embryos with CRISPR/Cas9 gene results in deletion of large fragments of DNA

In a new study, researchers from the University of Adelaide, Australia, the South Australian Institute of Health and Medical Research, La Trobe University, the Singapore-MIT Technology Alliance and the Temple University in the United States discovered a major obstacle to the realization of editing embryonic genes. The results of the study were published in the August 9th, 2018 issue of Nature.

Thomas said that their research provided another explanation for the genetic corrections achieved in that North American study: this gene editing technique did not fix small errors, but produced even bigger mistakes.

Thomas said, "Gene editing technology is still relatively new, and part of this research area includes understanding these deficiencies, which will ultimately enable us to develop the safest treatment for genetic diseases." Thomas and the first author of the paper, Fatwa Adikusuma, recreated the North American study using preclinical animal models. Australia has strict legislation to limit genetic editing of human embryos.

[4] Mol Cell: New research makes CRISPR gene editing safer and more accurate
The most important scientific advances in recent years have included the discovery and development of new ways to genetically transform organisms using a fast and affordable CRISPR technology. Now, in a new study, researchers from the University of Texas at Austin said they simply improved the technology, which would lead to more accurate and safer genetic editing. It is enough to safely open the door to genetic editing in the human body.

These researchers have found conclusive evidence that as the most popular enzyme currently used for editing CRISPR genes, also the first CRISPR protein to be discovered, Cas9 is less efficient and accurate in gene editing than a less used one - A CRISPR protein called Cas12a (formerly known as Cpf1). The results of the study were published online August 2, 2018 in the journal Molecular Cell.

[5] JACS: A new gene editing tool was developed- RIBOTAC
As scientists gain insight into which genes can contribute to disease, they are pursuing the next logical question: Can genetic modification techniques be developed to treat or even cure these diseases? Much of the research has focused on developing technologies such as CRISPR/Cas9, a protein-based gene editing system.

In a new study, Dr. Matthew D. Disney from the Chemistry Department of the Scripps Research Institute in the United States and his team took a different approach and developed a small molecule-based tool that works on RNA to selectively knock out certain gene products. This further confirms that RNA is indeed a viable target for the development of drugs.

The culling tool developed by the Disney team has made it possible to develop drugs that are used as pills to correct genetic diseases -- by destroying toxic gene products and chemically controlling the body's defense mechanisms. Related research results were published online May 24, 2018 in the Journal of the American Chemical Society.

[6] Nat Genet: Scientists improve genetic editing technology CRISPR to accelerate the editing of cell genomes

As a powerful gene editing tool, CRISPR can help scientists trim DNA with amazing precision, but tracking the effects of these changes on gene function is often time consuming, and researchers can only perform one analysis to one editing each time, and this process takes weeks.

Recently, in a research report published in the international journal Nature Genetics, scientists from the University of California, Los Angeles, through the improvement of CRISPR technology, achieved the goal of monitoring the results of tens of thousands of genetic editing. Related research can also improve the ability of scientists to identify genetic changes that often damage cells and cause disease.

Researcher Leonid Kruglyak said that scientists have been using CRISPR to cut multiple genes for many years. Currently, they still lack the method to edit multiple genes at the same time. A laboratory has developed a large-scale technology for the first time. This is done in cells that are structurally similar to human cells. Previously, the researchers performed parallel editing in bacterial cells. CRISPR can bind to the scrambled protein Cas9, which can be used as a guiding molecule to introduce CRISPR into a precise site. Once it reaches its destination, Cas9 begins to construct DNA to inactivate target genes. The researchers then insert new DNA fragments and edit the gene sequence, while also fixing the gap caused by Cas9.

Author's Bio:

This article is compiled by scientists from Creative Peptides. During the last decade, scientists in Creative Peptides have become increasingly involved with immunotherapy, cell therapy or gene therapy in an effort to combat cancer and other major diseases. To this end, many other techniques have also been employed, including Silver Nanoparticles Conjugation, bioconjugation, peptide nucleic acid synthesis, surface plasmon resonance imaging, Amino Acids Modification, neoantigen peptides vaccine synthesis, Epitope Mapping and more. For more information, please contact