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Gene-Editing Tool CRISPR-Cas9 Can Now Monitor And Target RNA In Living Cells

18 March 2016, 10:35 am EDT By Angela Laguipo Tech Times
For the first time, scientists used the CRISPR-Cas9 system on RNA in living cells. This will help treat certain diseases linked to a problem with RNA like autism and certain types of cancer.  ( David Nelles | University of California, San Diego. )

The new genetic tool called CRISPR-Cas9 can now be used as an accessible means to track and target the movement of RNA in living cells, researchers found.

A person's susceptibility to disease and characteristics like the color of the hair or eyes, are all encoded in the deoxyribonucleic acid or DNA. Scientists have long been sequencing human genome in order to develop ways to alter the genetic code. However, there are some diseases that are linked to RNA, another important molecule.

Researchers at the University Of California, San Diego figured out how to use the tool on the messenger RNA that carries genetic instructions from the nucleus of the cell to make new proteins.

"We are just beginning to see the implications of genome engineering using the CRISPR technology, but many diseases, including cancer and autism, are linked to problems with another fundamental biological molecule: RNA," said Gene Yeo of the University of California, San Diego.

"Future developments of this work could enable researchers to measure other features of RNA processing or support therapeutic approaches to correct disease-causing RNA behaviors," he added.

What Is The CRISPR-Cas9 System?

It's a naturally-occurring defense mechanism found in many bacteria wherein a strange pattern of DNA sequence would be repeated over and over again.

Some bacteria have two parts of its defense mechanism that protect them from vicious viruses that prey on them. Scientists found that the unique sequences in between the repeats were like that of the viruses' DNA.

They found that CRISPR is a part of the immune system of bacteria wherein it keeps parts of viruses so the next time these would attack; the bacteria can easily identify them. The second part entails having a set of enzymes called Cas or CRISPR-associated proteins. This can cut and slice invading viruses.

Otherwise known as the clustered regularly-interspaced short palindromic repeat, CRISPR-Cas9 is an RNA-guided gene-editing system derived from Streptococcus pyogenes. This is the most common type of Cas enzymes.

Tool Can Modify Genes To Treat Certain Illnesses

The scientists involved in the study conducted an experiment that could lead to new treatments for diseases that are caused by problems in the messenger RNA such as certain types of cancer, autism, neurodegenerative diseases and the fragile X syndrome.

The location of the RNA in cells can influence the production of proteins and determine whether they are created at the right time and place. When this transport fails, this can lead to a lot of conditions or diseases.

The scientists found that they could use the defense system, CRISPR-Cas9, for the first time since this has only been used to edit DNA. This can target RNA in living cells through a process they called RNA-targeted Cas9 or RCas9.

"DNA is the fundamental building block of life and we are just beginning to see the implications of genome engineering with CRISPR-Cas9, but many diseases including cancer and autism are linked to problems with another fundamental biological molecule-RNA," said David Nelles, a UC-San Diego graduate.

The study was published in the journal Cell.

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