Messenger RNA (mRNA) delivery to the liver corrects ornithine transcarbamylase deficiency in a mouse disease model

Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases

“…These results are highly promising, as no toxicity-associated side effects were observed, even upon multiple-dose application. 79 Besides OTC deficiency, other urea cycle deficiencies, e.g., argininosuccinate synthetase deficiency, are also likely to be treatable using mRNA-based approaches. As described by Heartlein, Derosa, and Smith, argininosuccinate synthetase protein levels could be detected already after a single i.v.…”