A new genome editing tool could revolutionise human gene editing, allowing us to modify our own genomes to improve our health and wellbeing.
The idea of gene editing is controversial because it relies on cutting DNA from other organisms, but scientists are confident that if it is implemented correctly it will eventually be feasible to achieve much more.
Researchers at the University of California San Diego, in collaboration with scientists from Imperial College London, have developed a tool that uses a special type of DNA called a plasmid, which is part of the cell’s genetic material, to deliver a small amount of genetic material to a target organism.
The researchers used the DNA from a mouse embryo to create a genetic copy of the human genome.
They then engineered the plasmids and used them to deliver the edited DNA directly to the mouse embryo.
To use this approach, researchers inject a small portion of the genetic material into a living animal and then deliver the genetic template into the animal.
After that, the modified gene is implanted into the embryo and the animal grows and develops normally.
However, scientists also tested a number of different approaches for gene editing in a mouse model.
In one experiment, researchers injected DNA from another mouse embryo into the same mouse embryo and then injected the modified plasmoid DNA into the mouse embryos.
They found that this approach was the most effective, but that it was not sufficient to replace all the DNA that was injected into the embryos, so they needed to inject a more complex DNA structure into the cells.
The team then injected a large amount of the modified DNA into a mouse and found that it could be delivered more effectively than the previous approach.
What’s more, they found that the DNA in the plasminogen activator, which acts as an anti-cancer gene, acted as a good guide for the engineered gene to find its way to the correct target organism, even when the plaits are not used to edit the target.
These results show that, with this new tool, we can achieve much higher efficiency than previous methods and are very promising for the future of gene-edited systems, said lead author Dr. Richard Schaller, an associate professor of biological sciences at UC San Diego.
“By creating this system, we have created an innovative and scalable tool that will be useful for future applications,” said Schallers co-author Dr. Daniel Nye, professor of biochemistry at Imperial College.
“We can now design a system that is much more efficient than the current generation of genome editing tools and that is designed to be highly stable.”
The new system also can deliver targeted DNA to a specific cell, rather than allowing the gene to be passed around between cells in the same organism, allowing scientists to modify the genome of a particular cell without damaging other cells.
The new method, called ‘RNAi’, can be injected directly into cells and then targeted to specific cells, allowing researchers to alter the genetic information that is stored in that particular cell.
This is important because if a gene is passed around to multiple cells, it is likely to affect their behaviour.
If the gene is inserted into cells with an altered DNA structure, it will be difficult to reverse the damage.
For this reason, the new system uses a plasmine called RNAi that is able to target DNA from the placenta and inject it into cells without damaging any cells.”RNAi was designed to target a particular gene, which in this case is the plastid-related gene p21, and deliver the desired gene directly into the target cell,” said Nye.
“Because the DNA is inserted directly into a target cell, we don’t have to worry about damage to other cells that may be carrying the gene.
We have already been able to do this for other gene editing tools that are already available, such as Cas9, which we developed in collaboration to deliver DNA directly into mouse embryos.”‘
Our work provides a powerful new tool for the next generation of gene cutting tools, and demonstrates that this is possible and is a significant step forward in gene editing research.’
The researchers are now working to improve the efficiency of the system, and hope that it will also be possible to improve efficiency and safety of this technology in future.
Their study was published in Nature Genetics.
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