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

Published 2015 View Full Article

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Title
Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification
Authors
Keywords
RNAi, GalNAc-conjugated siRNA, exosome, circulating RNA, heme biosynthetic disorder
Journal
Molecular Therapy-Nucleic Acids
Volume 4, Issue -, Pages e263
Publisher
Elsevier BV
Online
2015-11-04
DOI
10.1038/mtna.2015.36

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