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

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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Bibliographic Details
Main Authors: Amy Chan, Abigail Liebow, Makiko Yasuda, Lin Gan, Tim Racie, Martin Maier, Satya Kuchimanchi, Don Foster, Stuart Milstein, Klaus Charisse, Alfica Sehgal, Muthiah Manoharan, Rachel Meyers, Kevin Fitzgerald, Amy Simon, Robert J Desnick, William Querbes
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
RNAi
GalNAc-conjugated siRNA
exosome
circulating RNA
heme biosynthetic disorder
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116300488

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http://www.sciencedirect.com/science/article/pii/S2162253116300488

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