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Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS

Tetsuya Nagata, Chrissa A. Dwyer, Kie Yoshida‐Tanaka, Kensuke Ihara, Masaki Ohyagi, Hidetoshi Kaburagi, Haruka Miyata, Satoe Ebihara, Kotaro Yoshioka, Takashi Ishii, Kanjiro Miyata, Kenichi Miyata, Berit Powers, Tomoko Igari, S. Yamamoto, Naoto Arimura, Hideki Hirabayashi, Toshiki Uchihara, Hiroya Kuwahara, Takeshi Wada, C. Frank Bennett, Punit P. Seth, Frank Rigo, Takanori Yokota

2021Nature Biotechnology138 citationsDOI

Topics & Concepts

Gene knockdownOligonucleotideRNABlood–brain barrierGeneGene knockoutBiologySystemic administrationGene expressionCentral nervous systemMolecular biologyPharmacologyIn vivoEndocrinologyGeneticsRNA Research and SplicingRNA Interference and Gene DeliveryRNA regulation and disease
Cholesterol-functionalized DNA/RNA heteroduplexes cross the blood–brain barrier and knock down genes in the rodent CNS | Litcius