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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Publicado en:Mol Ther Nucleic Acids
Autores principales: 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
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2015
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Original Article
Acceso en línea: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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