Next-Generation Gene-Editing Technology Successfully Cures A Genetic Blood Disorder

Posted: Oct 27 2016, 11:59am CDT | by , in News | Latest Science News

Next-Generation Gene-Editing Technology Successfully Cures a Genetic Blood Disorder
A peptide nucleic acid developed at Carnegie Mellon is part of a gene editing system that has cured a blood disorder in mice. Credit: Carnegie Mellon University
  • Something Beyond CRISPR Technology able to Alleviate Hereditary Blood Disease in Rodents

It seems that something beyond CRISPR gene editing technology is able to alleviate a hereditary blood disease in rodents.

A latest state-of-the-art system has been used to cure a blood disorder in mice. Going beyond the CRISPR gene editing technology, this methodology alleviated the symptoms of the blood disease in rodents using an IV treatment. It was that simple.

It can be used in living animals and it erases genetic mutations. This method relied on peptide nucleic acid (PNA) molecules. This is a technology that has been pioneered at a prestigious institution.

After getting the FDA’s approval regarding the nanoparticles, they were used to deliver the PNA molecules side by side with the donor DNA to repair a faulty gene in mice.

CRISPR has failed to accomplished the same thing. CRISPR normally relies on DNA-cutting enzymes to perform operations on DNA and slice a gene or remove it.

There are two problems with this though. Firstly, the enzymes are too large to be administered to living bodies. Secondly, the enzymes often wreak havoc once they are inside the cells thus defeating their main purpose.

The novel method avoids both errors. The nanoparticles contain PNAs. PNA is designed in such a manner that it opens the double helix of DNA and works on the genes without any cutting activity.

This biotechnology could be used in the future to treat neurodegenerative diseases. The researchers used a PNA to target a faulty gene in beta thalassemia. This is a blood disease that decreases the production of hemoglobin.

The decreased hemoglobin causes a range of nasty symptoms. Once the operational basis of this technique was studied and performed in the lab, the scientists celebrated the new technology that even outdoes CRISPR.

The findings of this study reported in Nature Communications.

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<a href="/latest_stories/all/all/20" rel="author">Sumayah Aamir</a>
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