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Recent research advances on the CRISPR-Cas9

gene editing tool

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
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