Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria
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DOI: 10.1126/scitranslmed.abc0700
Abstract: Description Systemic and subcutaneous rApoAI-PBGD therapy protects against porphyrin precursor accumulation, pain, and motor neuropathy in AIP mice. Apoliprotein for enzyme deLIVERing Acute intermittent porphyria (AIP) is a metabolic disorder caused by loss-of-function mutations in… read more here.
Keywords: aip; acute intermittent; pbgd; mouse model ... See more keywords