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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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2015
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Original Article
On-line přístup: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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