Systemic messenger RNA as an etiological treatment for acute intermittent porphyria
Sign Up to like & getrecommendations! Published in 2018 at "Nature Medicine"
DOI: 10.1038/s41591-018-0199-z
Abstract: Acute intermittent porphyria (AIP) results from haploinsufficiency of porphobilinogen deaminase (PBGD), the third enzyme in the heme biosynthesis pathway. Patients with AIP have neurovisceral attacks associated with increased hepatic heme demand. Phenobarbital-challenged mice with AIP… read more here.
Keywords: systemic messenger; acute intermittent; intermittent porphyria; hpbgd mrna ... See more keywords