The latest Cell reports the new CRISPR:targeting RNA
Source: latest Cell reports the new CRISPR:targeting RNA
author:Chen Moisource: bio-explore
editor’s note
2018 opening year less than a quarter of the CRISPR field of the issued momentum no less! Recently, from the Salk biological Institute scientists in Cell journal has published an important outcome-targeting RNA of the CRISPR technology! Small note to this young team, there are many people with the“God”Zhang Feng related.
image source: Cell DOI: https://doi.org/10.1016/j.cell.2018.02.033）
3 on 15 May, published in the Cell Journal entitled“Transcriptome Engineering with the RNA-Targeting Type VI-D CRISPR Effectors”of the research, the Salk Institute forPatrick D. Dr. Hsu led the team that developed a new targeted RNA gene magic cut–CasRx and use it to correct from the dementia patient’s cells protein in imbalance, so that they are restored to healthy levels.
from DNA to RNA
CRISPRs are actually bacterial of the immune system, contain a variety of defensive enzymes, such as the well-known Cas9。 Based on Cas9, scientists have developed a targeted DNA of the gene editing tools.
Dr. Hsu said:“CRISPR has revolutionized genome engineering, genome engineering, andwe hope that can be this tool that can target to target, from DNA, to RNA.”
The researchers explained that a given RNA information can be in different levels of expression, it is relative to other RNA balance to healthy function is vital. In addition, the RNA can be through a variety of ways by splicing to produce different proteins; and the splicing process problems will lead to diseases, such as spinal muscular atrophy,atypical cystic fibrosis and frontotemporal dementia（frontotemporal dementia, FTD）it. Therefore, targeting“toxic RNAs”or“error splicing to generate the RNA”of the drug, is expected to suffer from these devastating diseases of the patients have a life-changing impact.
From left: Peter Lotfy, Silvana Konermann, Patrick Hsu, Jennifer Oki and Nicholas Brideau image source: Salk Institute
the best version-the CasRx
Based on this background, Dr. Hsu, etc. decided to continue to search the bacterial genome to find a can be targeted to the RNA of the new CRISPR enzyme.
The thesis of the first author Silvana Konermann, Dr. said:“started this project when we have such a hypothesis, i.e., in bacteria and other invading viruses in the race, the different CRISPR systems may have proprietary features, including the ability to target to the virus RNA.”
In the study, the scientists developed a computer program to search the bacterial DNA database to search for CRISPR system of signal: specific repeated DNA sequence. In this process,they found a target to RNA of the CRISPR enzyme family–Cas13d it.
image source: Cell
However, the research team realized that, like Cas9 family, like, from different kinds of bacteria Cas13d enzyme, its activity will vary, so continue the search,to find can be used for human cells the best version of Cas13d enzymes.ultimately, the scientists found thatfrom intestinal bacteria Ruminococcus flavefaciens XPD3002 the Cas13d enzyme is they want, and name it CasRx it.
CasRx (magenta) targeting RNA in the nucleus of human cells (gray). Image source: Salk Institute
effective rate of up to 80%
With the targeting of RNA to the other technologies compared to theCasRx is unique, because it is Size smallmakes it easier to be loaded into theTreatment-associated virus vector）, high availability, and with RNA interference compared to no significant off-target effects.
In neurodegenerative disease is FTD, the two versions of the tau protein ratio in the neurons lost in the balance. In this study, scientists genetically engineered a CasRx, so that its targeting over-expression of tau protein corresponding to the RNA sequence. They will CasRx packaging into a virus, and then delivered to the FTD patient’s stem cells to develop into neurons.< strong>the results show that CasRx adjust the tau protein to the health level of efficiency of 80%.
These results let Dr. Hsu, etc. feel very excited. He said:“nature is filled with so many secrets, it really is the invention of new technologies of rich resources.”
with Zhang Feng about
According to the Hsu Lab website presentation, Dr. Hsu worked at the Broad Institute and Zhang Feng together to work for the CRISPR-Cas9 technology for efficient and precise editing of the eukaryotic cell early in development to make a contribution; at the same time, he is also Zhang Feng, co-founder of Editas Medicine’s Lead Scientist, contributed to these gene editing techniques used in human genetic disease treatment.
image source: Science
In addition to Dr. Hsu, the previously mentioned Konermann Dr. also has worked with Zhang Feng to work with. She is Zhang Feng 2016 published an article critical Science paper on the figure of the co-authors.< strong>this paper first describes a method of targeting an RNA of the CRISPR enzyme-C2c2 now known as Cas13a it.papers published, Cas13a soon became CRISPR circle“new favorite”it. Up to now, scientists have published on the Cas13a number of breakthrough results, including the not long ago out of Jun introduced the upgraded version of diagnostic tool-SHERLOCK（detail:Feng Zhang the latest Science on! CRISPR a new feature upgrade that can be used for tumor DNA and other nucleic acid detectionit.
For the latest discovery, targeted RNA CasRx enzyme, Dr. Hsu team is also filled with anticipation. They believe thatCasRx having a genetically engineered RNA and protein of tremendous potential, it is the birth of researchers to develop novel gene therapy and the investigation of basic biological functions provides a powerful method.we look forward to this one magic clip new member of the family can be further bloom of its glory.
Original title: targeting RNA with! The latest Cell reports the new CRISPR, multiple authors with Zhang Feng about
reference:
CRISPR genetic editing takes another big step forward, targeting RNA
Published at Mon, 20 Mar 2018 22 :03:58 +0000
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- After ditching Editas, AbbVie taps Caribou for new CRISPR, CAR-T pact – FierceBiotech
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