Scientists Cure Blood Disorder in Mice Using New Gene Editing System

The novel system could be a better alternative to CRISPR.

10. 27. 16 by Jess Vilvestre
Image by AC/PsyPost

Better than CRISPR?

Move over, CRISPR. Scientists from the Carnegie Mellon University and Yale University have developed a new gene editing system, and they’ve used it to successfully cure a genetic blood disorder in living mice. The findings are published in Nature Communications.

The novel gene editing system takes a detour from the popular CRISPR gene editing technique, which has been used to treat similar diseases. Unlike CRISPR, which relies on cutting and pasting together genetic data surgically removed from target cells followed by reinsertion in the body, this system is non-invasive. It utilizes peptide nucleic acid (PNA) molecules, nano-sized synthetic molecules that look like a cross between proteins and nucleobases found in DNA and RNA. The PNA is able to open up DNA and deliver therapeutic genes for treatment.

“We have developed a system that uses FDA-approved nanoparticles to deliver our PNA molecule along with a donor DNA to repair a malfunctioning gene in living mice. This has not been achieved with CRISPR,” said Danith Ly, professor of chemistry in Carnegie Mellon’s Mellon College of Science and an expert in PNA chemistry.

Credit: Andy Wirth/Flickr

Ly, together with the other researchers, developed PNA to target the mutated gene in beta thalassemia, a blood disorder that manifests in reduced production of hemoglobin. The PNA was administered to mice with the blood disorder through intravenous injection. Their findings report a 7 percent successful gene editing in the blood stem cells and an increase of hemoglobin level for several months post-treatment. This is a major step up from the 0.1 percent success rate of typical gene editing methods.


New Cures on the Way

As Ly mentioned, PNA is already FDA-approved and has previously been used to treat neurodegenerative diseases in humans, which means there are fewer barriers to use for this team’s gene editing system.

Gene editing is a continually maturing field, and we’re discovering more and more ways on how to use it to improve our lives. It could help us find a cure for HIV, fight famine, and maybe even beat cancer. As more research is conducted, the technology will only get better.

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