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Delivery of oligonucleotides to the liver with GalNAc: From research to registered therapeutic drug.

Debacker, Alexandre J., Voutila, Jon, Catley, Matthew, Blakey, David, Habib, Nagy·2020·Molecular Therapy
DOI 10.1016/j.ymthe.2020.06.015
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  • Specific targeting of hepatocytes can be achieved using GalNAc-conjugation, such as GalNAc-siRNA or antisense oligonucleotides, which has been successfully used to silence hepatic PCSK9 expression in a clinical trial.

    “Specific targeting hepatocytes can be archived using N-acetylgalactosamine (GalNAc)-conjugation, such as GalNAc-siRNA or antisense oligonucleotides (ASOs) (50–52), which has been successfully used to silence hepatic PCSK9 expression in a clinical trial (51).”

    From Identification of amino acid residues in the MT-loop of MT1-MMP critical for its ability to cleave low-density lipoprotein receptor (2022) · cites this paper as [52]

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Research works printed but not linked: 77

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Metadata

Authors
Debacker, A. J., Voutila, J., Catley, M., Blakey, D., & Habib, N.
ORCID
Debacker, Alexandre J.
Venue
Molecular Therapy
Publisher
Elsevier BV
Published
2020-08
Volume
28
Issue
8
Pages
1759-1771
ISSN
1525-0016
DOI
10.1016/j.ymthe.2020.06.015
Cited by
424
References
11
Language
en
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