![]() Patients with this disease, which affects about 1 in 100,000 people, cannot break down the amino acid tyrosine, which accumulates and can lead to liver failure. Current treatments include a low-protein diet and a drug called NTCB, which disrupts tyrosine production. In experiments with adult mice carrying the mutated form of the FAH enzyme, the researchers delivered RNA guide strands along with the gene for Cas9 and a 199-nucleotide DNA template that includes the correct sequence of the mutated FAH gene. Using this approach, the correct gene was inserted in about one of every 250 hepatocytes - the cells that make up most of the liver. Over the next 30 days, those healthy cells began to proliferate and replace diseased liver cells, eventually accounting for about one-third of all hepatocytes. This was enough to cure the disease, allowing the mice to survive after being taken off the NCTB drug. ![]() “We can do a one-time treatment and totally reverse the condition,” says Hao Yin, a postdoc at the Koch Institute and one of the lead authors of the Nature Biotechnology paper. “In particular, the authors note that the efficiency of gene editing will need to improve significantly to be relevant for most diseases and other delivery methods need to be explored to extend the approach to humans. Nevertheless, this work is an exciting first step to using modern gene-editing tools to correct the devastating genetic diseases for which there are currently no options for affected patients.” “This work shows that CRISPR can be used successfully in adults, and also identifies several of the challenges that will need to be addressed moving forward to the development of human therapies,” says Charles Gersbach, an assistant professor of biomedical engineering at Duke University who was not part of the research team.
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