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Successful Correction of Genetic Problems In Mice Before Birth Raises Hopes of Similar Treatments For Humans

An anonymous reader quotes a report from The Guardian: Gene editing to correct faulty DNA in human embryos has taken a step closer to becoming a reality, with scientists showing it is possible to correct genetic problems in mice before they are born. Researchers used a form of the gene-editing tool Crispr-Cas9 to introduce a mutation into a gene that would otherwise cause lethal liver failure in mice. While the approach has previously been shown to work in mice after birth, the latest study showed it was also possible to make the all-important tweak before they were born. Writing in the journal Nature Medicine, a team of researchers in the US report how they conducted a series of experiments to explore the use of gene editing in mouse fetuses using a modified form of Crispr that can alter single “base pairs” — the molecules that couple up to form the rungs of the DNA double helix — but only cuts one strand of DNA when making a change.

After showing it was possible to make a change at a particular spot in the DNA of liver cells in mouse fetuses, the team focused on a condition known as hereditary tyrosinemia type 1. This is a genetic disease that prevents the body from breaking down an amino acid called tyrosine as it should, and can cause death if left untreated. The team took mice with a genetic mutation that produced a similar condition to hereditary tyrosinemia type 1 and bred them, with the mothers kept on a drug called nitisione. The team then injected 26 of the fetuses with a virus carrying the genetic instructions for making the gene-editing tool, and 27 of them with the same virus but without information for the tool. After birth the baby mice no longer received the drug, and the team watched what happened. “In the non-treated mice, they all died by 21 days of life,” said Dr William Peranteau, a pediatric and fetal surgeon at the Children’s Hospital of Philadelphia who co-led the study. “However, those that had been treated were able to survive until the end of the study at three months, and looked very similar to another group that had not been injected with anything and were kept on the drug. No gene editing was seen in the mothers who had given birth to the mice.

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