Researchers have succeeded in correcting a gene defect that causes a hereditary liver disease and its adverse effects on cells.
Researchers at the University of Helsinki and HUS Helsinki University Hospital have succeeded in correcting a gene defect that causes a hereditary liver disease and its adverse effects on cells.
Researchers from the University of Helsinki and HUS Helsinki University Hospital have succeeded in correcting the gene defect associated with argininosuccinic aciduria and demonstrated that the harmful metabolism caused by the disease can be cured. "This 'gene mixture' we produced is based on the formula of a pharmaceutical product already in use, which may facilitate its clinical use in the future. Our next goal is to cure ASLD in mice," says Doctoral Researcher Timo Keskinen from the University of Helsinki.
"However, a cure could be possible if the gene defect causing the disease is eliminated entirely. Thanks to basic research carried out with the help of gene scissors and other precise gene-editing techniques, permanent fixes are gradually starting to emerge," Wartiovaara says.
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