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What if researchers could turn a diseased cell into a healthy one by simply reprogramming its genes? Using a type of adeno-associated virus, a popular tool in gene therapies, a team at the University of California, San Francisco was able to transform cirrhosis-causing liver cells into normal ones within the body of a mouse. Study leader Milad Rezvani says that adding healthy cells to a diseased liver is not an easy task, so the prospect of gene therapy offers a safe and possibly more efficient alternative.
"Diseased livers are actually not a good target for cells to be transplanted – they don’t engraft well."
A gene delivery vehicle like a virus provided a way to do this without surgery or cell transplantation.
"They can be used because they are safe. These are so-called adeno-associated viruses, which are gene vehicles packaged in viruses that do not integrate into the host genome. As a matter of fact, there are ongoing clinical trials using the kind of virus that we used." |