Intravenous administration of an mRNA for hPBGD encapsulated in Mtx‐LNPs resulted in high protein expression and enzyme activity in AIP mouse livers in less than 2 h and in full protection against acute porphyria attacks with a highly significant reduction in pain and motor neuropathy.
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The sentences
Treated animals also showed a trend toward a decrease of blood ammonia and a significant reduction of sucrose aversion, a hallmark of citrin deficiency.