Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates

Therapeutics based on short interfering RNAs (siRNAs) delivered to hepatocytes have been approved, but new delivery solutions are needed to target additional organs. Here we show that conjugation of 2′- O -hexadecyl (C16) to siRNAs enables safe, potent and durable silencing in the central nervous sy...

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Bibliographic Details
Published in:	Nature biotechnology Vol. 40; no. 10; pp. 1500 - 1508
Main Authors:	Brown, Kirk M., Nair, Jayaprakash K., Janas, Maja M., Anglero-Rodriguez, Yesseinia I., Dang, Lan T. H., Peng, Haiyan, Theile, Christopher S., Castellanos-Rizaldos, Elena, Brown, Christopher, Foster, Donald, Kurz, Jeffrey, Allen, Jeffrey, Maganti, Rajanikanth, Li, Jing, Matsuda, Shigeo, Stricos, Matthew, Chickering, Tyler, Jung, Michelle, Wassarman, Kelly, Rollins, Jeff, Woods, Lauren, Kelin, Alex, Guenther, Dale C., Mobley, Melissa W., Petrulis, John, McDougall, Robin, Racie, Timothy, Bombardier, Jessica, Cha, Diana, Agarwal, Saket, Johnson, Lei, Jiang, Yongfeng, Lentini, Scott, Gilbert, Jason, Nguyen, Tuyen, Chigas, Samantha, LeBlanc, Sarah, Poreci, Urjana, Kasper, Anne, Rogers, Arlin B., Chong, Saeho, Davis, Wendell, Sutherland, Jessica E., Castoreno, Adam, Milstein, Stuart, Schlegel, Mark K., Zlatev, Ivan, Charisse, Klaus, Keating, Mark, Manoharan, Muthiah, Fitzgerald, Kevin, Wu, Jing-Tao, Maier, Martin A., Jadhav, Vasant
Format:	Journal Article
Language:	English
Published:	New York Nature Publishing Group US 01.10.2022 Nature Publishing Group
Subjects:	631/154/152 692/308/2778 Agriculture Alzheimer's disease Amyloid precursor protein Bioinformatics Biomedical and Life Sciences Biomedical Engineering/Biotechnology Biomedicine Biotechnology Central nervous system Conjugates Conjugation Dosage Gene expression Gene silencing Hepatocytes Intranasal administration Life Sciences Lipophilic Lipophilicity Lungs Neurodegenerative diseases RNA-mediated interference siRNA
ISSN:	1087-0156, 1546-1696, 1546-1696
Online Access:	Get full text
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