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

• Glavni autori: Amy Chan (Autor), Abigail Liebow (Autor), Makiko Yasuda (Autor), Lin Gan (Autor), Tim Racie (Autor), Martin Maier (Autor), Satya Kuchimanchi (Autor), Don Foster (Autor), Stuart Milstein (Autor), Klaus Charisse (Autor), Alfica Sehgal (Autor), Muthiah Manoharan (Autor), Rachel Meyers (Autor), Kevin Fitzgerald (Autor), Amy Simon (Autor), Robert J Desnick (Autor), William Querbes (Autor)
• Format: Knjiga
• Izdano: Elsevier, 2015-01-01T00:00:00Z.
• Teme: article
• Online pristup: Connect to this object online.

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...