Scientists have raised fresh hopes for treating people with genetic disorders by inventing a powerful new molecular tool that, in principle, can correct the vast majority of mutations that cause human genetic diseases. From a report: The procedure, named “prime editing,” can mend about 89% of the 75,000 or so harmful mutations known to mangle the human genome and lead to conditions such as cystic fibrosis, sickle cell anaemia, and a nerve-destroying illness called Tay-Sachs disease. The landmark work opens the door to a new era of genome editing, but scientists caution that more research is needed before it can be safely used in humans. Beyond proving its safety, another major hurdle is how to deliver the molecular machinery to cells that need it in sufficient amounts to treat a disorder.
“This first report of prime editing is the beginning rather than the end of a longstanding aspiration in the life sciences to be able to make any DNA change in any position of a living cell or organism, including potentially human patients with genetic diseases,” said David Liu at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. The ability to rewrite the genetic code is one of the most striking scientific advances of recent years. The most common approach, known as Crispr-Cas9, has been likened to “molecular scissors” which home in on a particular DNA sequence and then cut it in two. The procedure allows scientists to disable specific genes and even correct harmful mutations by providing cells with fresh strands of DNA with which to repair the cut.
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