Posted by on June 25, 2020 9:33 am
Categories: Uncategorized

2 years 8 CRISPR/Cas9 security risks papers published,how to look at the “universal magic shears”

with the zinc finger nucleic acid enzyme of zinc finger nuclease, ZFN, a class of transcription-activating factor effect of the nucleic acid enzyme transcription activator-like effector nuclease, TALEN, and clustered regularly spaced Short Palindromic Repeat sequence Clustered regularly interspaced short palindromic installed or support, CRISPR and the single-base editor base editor and other techniques have emerged, the human turned on a gene editing“Pandora’s box”. span>

figure Shu gene editing technology development and applications source: papers

early in the 20th century, people began to gene editing technology to explore, but until 2013, the CRISPR/Cas9 technology successfully used in mammalian cells, was greatly facilitated gene editing technology for the development of the boom. Cell therapy and gene therapy is also considered to be within the next ten years, the field of life Sciences hot spot direction.

“hot spot theory”is not groundless, the capital of the “fanaticism” and the enterprise quickly into the Bureau is to signal the release of the beginning, but the development of more than about CRISPR/Cas9 underlying issues also gradually surfaced.

CRISPR/Cas9“Golden Wind”has been blowing up five years

ever since the 20th century 50’s discovered the structure of DNA since the human race has managed to learn how to “read” the genetic code, but with the CRISPR/Cas9 Discovery, people learn how to “write” it. Since in 2013 it was found that since the CRISPR’ve got many new applications, more and more companies are using this unique and powerful tool to solve complex problems. CRISPR-accurate editing of the genetic code, the ability is very powerful, and versatile, the outside world generally believe that CRISPR/Cas9 will be the future promises to revolutionize health care, agriculture and clean energy industry development pattern.

nearly five years, with the gene editing technology is gradually Mature, the CRISPR/Cas9 scenarios more diverse, and overseas companies on the CRISPR/Cas9 gene editing technology to the layout of the large screen also thus opened.

CRISPR gene editing technology is mainly R & D personnel, known as “CRISPR mother” Jennifer Doudna in 2017, leading the creation of a Mammoth Biosciences company, the use of CRISPR unique ability to accurately discover and bind to specific DNA sequences. The company to create the first CRISPR-mediated human disease detection platform, can achieve immediate and cost-effectively with disease-related RNA / DNA sequence for multiple testing.

and another CRISPR/Cas9 one of the founders of the Massachusetts Institute of Technology Professor Feng Zhang and also colleagues co-founded a biotechnology company Beam Therapeutics, designed by targeting a specific base to improve CRISPR accuracy.

more than that, the domestic and foreign enterprises on the CRISPR/Cas9 mining is still in the gradually in-depth.

source: the brilliance according to the disclosure of information does not completely finishing)

diamonds according to public information to incomplete statistics, summary and are summarized in the CRISP/Cas9 technology for the main direction of the enterprise. From the above table it is easy to see in the 2015-2020 years this 5 years, the CRISPR/Cas9 technology development speed rapid, the enterprise incoming enthusiasm unabated.< span class="bjh-strong">in genetic disease treatment, disease monitoring, cancer drug development, personalized medicine guidance, energy, agriculture and food, such as the direction are covered, visible CRISPR/Cas9 scope of application is not confined to a single field, but showing the package Luo Vientiane trend.

security risks it? To dialectically see

however, the“sweet melon bitter the pedicle, there was no America”, the CRISPR/Cas9 tool“is not perfect”. Since its birth, about the CRISPR/Cas9 off-target of the discussion has not stopped, as the research gradually in-depth, more safety problems are also among the many papers study be disclosed.

source: the brilliance according to the disclosure of information does not completely finishing, click on the picture to View Details)

according to finishing, the diamonds found in academic studies, around the CRISPR/Cas9 tool safety hazards of the research there are mainly 5 Direction, 1 triggered DNA damage; 2)throws enrichment of mutations; 3 with carcinogenic potential; 4)the broken ring chromosome stability; 5) The body of the Cas9 protein in the presence of an immune response, etc.

universal magic cut is no longer“a panacea”for? In this regard, Insiders on FAI said,“I think that few issues are more important, but the CRISPR/Cas9 technology itself has many different forms, different forms produce different problems, and the extent of the problem is also different.”

to-base editing tool, for example, he believes that the base editing tool, the Cas9 protein requires extra links active enzymes, mostly from bacteria and other microorganisms and the human body extract and through the engineered protein, which lead to body immune response and the unknown resistance is greater, the risk will be slightly higher.“ Even if the Cas9 protein itself, the presence of the issues have been resolved, but as the clip tool part associated therewith and the activity of the protein tools produced off-target effects, immune response and toxicity, etc. will give a base editing tool used to bring certain risks.”

to this he added that different tools have their own characteristics, at this stage, on gene editing tool more types of the premise, the use of tools to personalized medicine, the need for tools for assessment of balance functional value and safety issues.“ The tool itself is concerned, to do your evaluation on.”

secondly,his life FAI said, different disease the treatment will produce a specific risk degree of risk there are also cell therapy, for example, a gene editing tool in cell therapy of the application process, the operation of multiple in vitro completed, also during the presence of uncertainty, the need for operation of the process for quality control and screening.

for in vivo gene therapy, the tool is delivered into the body of the procedure need by means of viral vectors or non-viral vectors to complete, during the generation of the security issues and cell therapy different. At this stage, the cells treatment mainly for cancer, and in vivo gene therapy is mainly a genetic disease, the indications for the different, the risk also changes.“ For cancer patients in terms of whether the patient or family members are more willing to tolerate or bear certain risks, but for patients suffering from genetic disease of children, the Cas9 protein if there is a potential risk of cancer, will be on the CRISPR/Cas9 tool used to bring no small challenge.”

integrated tools, treatment modalities and disease these three differences in alienation factors, the professionals on Hui said,is now more like the personalized medical like to discuss gene editing tools the impact of dialectical integration of the respective advantages and potential risks.“not a single to demonstrate‘good’or‘bad’is.”

on gene editing tool different from the chromosomal damage, the participants believe that the traditional gene editing tools and single-base editing tool Base editor will bring a certain amount of DNA damage, in relative terms, the single-base editing tool damage is relatively small.

in addition to the above security issues, he is also on Hui said,CRISPR/Cas9 there is still a need to address the limitations and challenges: delivery and immune��Should be.

he believes that the current in the dissertation research, through the delivery of tools to transform can really get a good effect, but the real gene editing tools for clinical practice, the delivery efficiency may be greatly reduced, is still in the continuous optimization stage. At the same time,for a tool to elicit an immune response research is currently still relatively small, because of the need to through the clinical trials and data to the immune response to expand the system to study.

“that is why now gene therapy companies are in the advance Phase I clinical trials, because only into the clinical, to go with the facts speak.”

Published at Fri, 25 Jun 2020 13:32:25 +0000