Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

The acute hepatic porphyrias are caused by inherited enzymatic deficiencies in the heme biosynthesis pathway. Induction of the first enzyme 5-aminolevulinic acid synthase 1 (ALAS1) by triggers such as fasting or drug exposure can lead to accumulation of neurotoxic heme intermediates that cause disea...

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Bibliografiske detaljer
Udgivet i:Mol Ther Nucleic Acids
Main Authors: Chan, Amy, Liebow, Abigail, Yasuda, Makiko, Gan, Lin, Racie, Tim, Maier, Martin, Kuchimanchi, Satya, Foster, Don, Milstein, Stuart, Charisse, Klaus, Sehgal, Alfica, Manoharan, Muthiah, Meyers, Rachel, Fitzgerald, Kevin, Simon, Amy, Desnick, Robert J, Querbes, William
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group 2015
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4877445/
https://ncbi.nlm.nih.gov/pubmed/26528940
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2015.36
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