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Gene editing uses HIV virus to fight disease

By Sarah Curran Ragan     Jun 19, 2014 in Science
A new technology has been developed that uses the HIV virus as a weapon against hereditary infection. In the longer term it will also fight HIV infection say the researchers.
The growing field of genetic engineering has had many attempts to alter the genetic code of an organism to allow certain “desirable” traits and prevent “undesirable” ones. It's a method commonly used in selective breeding of livestock and in agriculture. And genetic research routinely uses so called “gene editing” whereby a gene is turned off to identify its role.
Disease caused by gene mutations would especially benefit from sophisticated technology now available for gene editing as it can also replace faulty genes. However its safety in humans has been difficult to secure. A virus used to “deliver” a new gene into DNA can prove problematic, removing more genes than intended.
Now a new study has used the HIV virus to solve these problems. The work has benefited from many years of research into HIV, showing that HIV particles can be used to transport genetic information into cells, say the authors. HIV infection is one of the areas the researchers are particularly keen to use the new technique.
The team from Denmark's Aarhus University, have developed a “hit and run” method for getting the customized enzymes, or nucleases, that carry the virus into cells, to a stay active for only a short time.
The method uses a virus to deliver two different nucleases to a cell. Once inside the cell, the viruses are able to release the nucleases removing a quarter of their gene targets. There were few errors reported in the results.
This is the first time scientists have been able to alter HIV virus particles to cut and paste into the human genome and it makes repairing genes more possible in new a safer ways.
It offers good prospects for the treatment of hereditary and many viral diseases such as HIV say the authors.
“By altering relevant cells in the immune system (T-cells) we can make them resistant to HIV infection and perhaps at the same time equip them with genes that help fight HIV” Yujia Cai, lead author of the study, says in a press release.
In the same study, the team developed a technique that improves the safety of gene editing. Once the gene targets were removed, the team added “gene patches,” new genes to replace those removed by the nucleases, to the viruses. The cut and patch strategy was a success in eight percent of treated cells.
“In the past the gene for the scissors has been transferred to the cells, which is dangerous because the cell keeps producing scissors which then start cutting uncontrollably,” says Associate Professor Jacob Giehm Mikkelsen, of the research team, in a press release.
But, because the team made the scissors in the form of a protein, they only cut for a few hours, after which they are broken down. This ensures that the virus particle brings a small piece of genetic material with it to patch the hole.
The work is a world first, “we can now simultaneously cut out part of the genome that is broken in sick cells, and patch the gap that arises in the genetic information which we have removed. We bring the scissors and patch together in the HIV particles in a way no one else has done before”
Targeted genome editing by lentiviral protein transduction of zinc-finger and TAL-effector nucleases
Yujia Cai, Rasmus Bak, Jacob Giehm Mikkelsen.Aarhus University, Denmark -
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