A mutation in the child’s liver cells had led to a potentially fatal disorder but medics, using CRISPR technology, were able to correct it.
CRISPR – short for Clustered Regularly Interspaced Short Palindromic Repeats – enabled doctors to target the faulty gene with an enzyme called Cas9. The enzyme makes a cut in the mutated gene and the body’s natural defence mechanism kicks in to repair it – and make the DNA as good as new.
“It seems to me to be a scientific ‘miracle’ that has made it possible to cure a very rare severe disease, and provides knowledge to treat many other diseases,” she added.
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It causes ammonia to build up to toxic levels in the body, which can cause brain damage and organ failure.
The baby, who has not been named, also coped well with a gastrointestinal illness that would normally be extremely dangerous to a child with their condition.
According to the study, the treatment is ongoing but researchers are “cautiously optimistic about the baby’s progress.”
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“Individualised, tailor-made therapies for a single patient are no longer a dream,” he said.
The research team said the success of the procedure “opens the possibility of creating personalized treatments in other parts of the body”.
“Although this has been a very specific approach, partly motivated by the devastating nature of the disease, it represents a milestone that demonstrates that these therapies are now a reality.
He added: “In summary, this work is proof of principle for a rapid and effective protocol for CRISPR-Cas therapies for the cure of human diseases in general and so-called rare diseases in particular, opening the door to other similar treatments in the near future.”
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