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🔬 An RNA-guided system in animals shows potential for gene editing in humans

🔬 An RNA-guided system in animals shows potential for gene editing in humans

  • A new programmable RNA-guided system, based on a protein called Fanzor, has been discovered in eukaryotes.
  • Bacteria use “scissors” called CRISPR-Cas against viruses, and now scientists have discovered that complex cells, like our own, also have a similar tool.
  • Fanzor systems can be delivered to cells more easily than CRISPR-Cas systems.

Discovery of a new gene editing mechanism

doctor. Feng Zhang’s team, known for its contributions to the field of CRISPR research, recently presented the first RNA-guided system in eukaryotes. This may have a significant impact on the future of gene editing in humans.

published in natureThe research shows how Fanzor proteins target DNA using RNA guides. They demonstrated Fanzor’s ability to edit the human genome more precisely. This compact system may be more suitable for treatment than current CRISPR-Cas systems.

While CRISPR-Cas was first identified in simpler organisms, Zhang and his team demonstrated the existence of RNA-guided DNA-cutting mechanisms in eukaryotes—organisms that include fungi, plants, and animals.

“This new system is another way to make subtle changes in human cells, complementing the gene-editing tools we already have,” says Chang.

doctor. Feng Chang. Photo: Justin Knight, McGovern Institute

Discovery explained

  • Natural defense: Just as our immune system helps us fight infections, bacteria have a system called CRISPR-Cas to defend themselves against viruses. They use these “scissors” to cut the DNA of the invading virus.
  • New discovery in eukaryotes: Scientists knew bacteria had these “scissors,” but they weren’t sure if more complex cells (such as human cells) had them, too. We now know that more complex cells also have a similar tool.
  • Genetic Engineering: Knowing about these “scissors” is exciting because they can be used as tools for gene editing. Imagine the possibility of correcting defective genes that cause disease.
  • Wherever there is life: The findings suggest that RNA-guided “scissors” are so important that they are found in all forms of life, from tiny bacteria to humans.
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From bacteria to complex life forms

Understanding the building blocks of life was a major goal of Zhang’s lab. Their mission is to develop gene medicines that target specific genes and processes. They previously discovered programmable RNA systems in prokaryotes called omegas. This led to the feeling that Fanzor enzymes in eukaryotes could also utilize RNA-guided mechanisms.

picture: Chang Lab, the MIT Broad Institute and the Harvard/McGovern Institute for Brain Research at MIT.

Unlike CRISPR proteins, Fanzor enzymes are encoded within ‘jumping’ genes or transposable elements from eukaryotic genomes. This indicates that these genes have been transferred between bacteria and eukaryotes over time.

Fanzor’s potential as a gene editing tool has been demonstrated in human cells. And although it was initially less effective than CRISPR-Cas, scientists have made progress increasing its activity tenfold. The main advantage is that Fanzor does not cause ‘overtime’, which reduces the chance of unwanted genetic modifications.

Nature’s Comprehensive Toolbox

Zhang’s team believes this discovery may be the tip of the iceberg.

“Nature is amazing. There’s so much diversity,” says Zhang, noting that more programmable RNA systems could be found.

This research opened up a new possibility for gene editing. While CRISPR remains a powerful tool, Fanzor’s discovery and its potential offer an alternative route, including easier delivery of cells.

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