Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing
Nair JK, Willoughby JLS, Chan A, Charisse K, Alam MR, Wang Q, et al. · Journal of the American Chemical Society, 2014
Nair JK, Willoughby JLS, Chan A, Charisse K, Alam MR, Wang Q, et al.
The delivery breakthrough that made siRNA drugs practical. Attaching a three armed GalNAc sugar to an siRNA lets liver cells take it up efficiently after a simple injection under the skin, giving durable gene silencing at low doses. GalNAc conjugation is now the backbone of most approved and clinical siRNA medicines.
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