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Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug

Targeted delivery of oligonucleotides to liver hepatocytes using N-acetylgalactosamine (GalNAc) conjugates that bind to the asialoglycoprotein receptor has become a breakthrough approach in the therapeutic oligonucleotide field. This technology has led to the approval of givosiran for the treatment...

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Bibliografiska uppgifter
I publikationen:Mol Ther
Huvudupphovsmän: Debacker, Alexandre J., Voutila, Jon, Catley, Matthew, Blakey, David, Habib, Nagy
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2020
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7403466/
https://ncbi.nlm.nih.gov/pubmed/32592692
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.06.015
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