Imagine being able to correct genetic defects with the precision of a surgeon. Thank you to the new ones compact molecular scissorsthis vision is closer to reality than you think. Recently, researchers have developed a smaller, more efficient version of molecular scissors for the DNA cuttingovercoming the limitations of the well-known Crispr-Cas9 system. These scissors, based on bacterial protein TnpBpromise to revolutionize genetic medicine.
What is the TnpB protein and how does it work?
There TnpB it’s one derived protein from the bacterium Deinococcus radiodurans, known for its incredible resistance to extreme conditions, so much so that it was nicknamed “Conan the bacterium”. In this study, published on Nature Methodsresearchers from the University and ETH Zurich have engineered this small protein, improving its efficiency up to four times in cutting DNA. But what makes TnpB so special?
Unlike the more well-known system Crispr-Cas9which requires high doses and more viral particles to be delivered into cells, the TnpB system can be packaged into a single virus particle. This makes the DNA modification simpler and more efficient, especially for interventions on organs such as the liver and brain.
A success against familial hypercholesterolemia
The first results on mice are extraordinary: the new molecular scissors were able to correct the genetic defect responsible forfamilial hypercholesterolemiaan inherited condition that causes high levels of cholesterol in the blood. In tests on mice, this new technology reduced cholesterol levels by a significant amount 80%! This could pave the way for future gene therapies for humans.
The role of artificial intelligence
The discovery of these new molecular scissors would not have been possible without the help ofartificial intelligence. The researchers tested TnpB on more than 10,000 target sites, using an AI model to predict the efficiency of the gene editing system in different scenarios. Thanks to these predictions, the tests achieved an efficiency of 75.3% in the liver and of 65.9% in the brain of mice.
One of the main advantages of TnpB compared to Crispr-Cas9 is its compactness. Being much smaller, TnpB can be packaged into a single viral particle, making the DNA editing process faster and less complex. On the contrary, Crispr-Cas9 requires more viral particles to be transported, increasing the risks and costs associated with therapy.
Furthermore, the TnpB has demonstrated greater precision in recognizing the exact point at which to cut the DNA, reducing the risk of side effects or unwanted changes in the genome.
The future of gene editing
This new technology could pave the way for safer and more accessible treatments for a wide range of genetic diseases, including hypercholesterolemia, cystic fibrosis and other inherited conditions. Scientists are already working to further improve these molecular scissors and test them in other animal models, with the hope of starting clinical trials in humans in the coming years.
Isn’t it fascinating to think that thanks to small bacterial proteins we could soon correct genetic diseases that afflict millions of people around the world?
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