Two research results that advance genetic editing based on "CRISPR" will be announced at nearly the same time

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New techniques were announced in a series of ways by editing information contained in genes. One of them is a new method using "CRISPR-Cas9 system" and the other one is to edit RNA, which has been announced by the same research institution.

New gene-editing technique may lead to treatment for thousands of diseases - LA Times
http://www.latimes.com/science/sciencenow/la-sci-sn-dna-gene-editing-20171025-story.html

'Incredible' editing of life's building blocks - BBC News
http://www.bbc.com/news/health-41724994

These technologies were announced by the research institution "Broad Institute" jointly operated by Harvard University and Massachusetts Institute of Technology. Currently, "CRISPR-Cas 9", which cuts out a part of the genome containing genetic information and replaces it with another genome, is the center of genetic editing technology, but the new method can be said to have further advanced this Has become.

◆ Rewriting the base of genes directly "Base editing"
The DNA, which is the blueprint for the organism, contains deoxyribose, phosphoric acid, and bases. The base is further divided into four groups: adenine (A), guanine (G), cytosine (C), thymine (T), but "base editing" which edits genetic information by editing these bases ) Has been developed as a method called.

Programmable base editing of A • T to G • C in genomic DNA without DNA cleavage | Nature
https://www.nature.com/articles/nature24644.epdf


If the past gene editing is to cut and paste a certain mass using scissors, the newly announced method is like directly rewriting the character of the gene using a pencil and an eraser. Both methods utilize the CRISPR - Cas 9 system, which is the core of genetic editing technology.

In the newly announced method, we first search and capture the desired gene sequence using CRISPR-Cas9. Here, in the past, the sequence of the gene was cut off and replaced with a new sequence, but in the newly announced method, the DNA strand where the genomic information is stored is separated, and using the enzyme attached to the CRISPR system, It is a mechanism to rewrite the base to be correct.

Professor David Liu of Broad Institute says, "We are advancing research to apply base editing technology to the treatment of human body." But there seems to be still a challenge, Professor Liu said, "It is the first step to complete the mechanism to edit bases is the first step, although there are still things to do, this mechanism will be very useful" I am talking.

◆ How to rewrite RNA
If DNA is a "design drawing" that stores information on individuals, RNA can be said to have the function of copying the information and taking it out and carrying the necessary protein. The research team tried to edit genes that cause hereditary anemia by using this approach to RNA.

RNA editing with CRISPR-Cas 13 | Science
http://science.sciencemag.org/content/early/2017/10/24/science.aaq0180


Here, the research team uses a technique that uses a different nuclease "Cas 13" than CRISPR-Cas 9. Feng Zhang of the Broad Institute said, "Correcting disease-causing mutations is one of the main objectives of editing genomic information.This way to enable RNA editing occurs in almost all cells We will have the potential to be able to treat many diseases. "

Dr. Helen O'Neill of University College London said that these papers were announced the same week, "It has become an interesting week in gene research and these papers are rapid evolution in the field of genetic editing It is a highlight of speed and uninterrupted progress, and we are coming closer to the actual medical level step by step. "

Also, Dr. Sarah Chan at the University of Edinburgh said: "Science is progressing at a tremendous speed, has become less dangerous, has become more reliable, accurate and effective, and no longer has a lot of opinions from people on genetic editing The ages such as aggregating the age are totally in the past, "he says of a situation that can not be avoided as" genetic editing is dangerous "as it has been.

There are also many diseases caused by genes, there are 50,000 cases in which diseases occur due to mutation of genes, and cases that become sick just by rewriting one of the bases constituting the gene Thirty thousand 2000 cases have been confirmed. By establishing and putting into practical use the gene editing technology announced this time, it is expected that the disease caused by the "misprint" of genes will be greatly reduced.