Delivery

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

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Journal of the American Chemical Society2014Delivery

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.

Source

Journal of the American Chemical Society, 2014. DOI: 10.1021/ja505986a

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