{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["29(10)"],"submitter":["Holland RJ"],"pubmed_abstract":["N-Acetylgalactosamine (GalNAc) conjugated short interfering RNAs (siRNAs) are a leading RNA interference (RNAi) platform allowing targeted inhibition of disease-causing genes in hepatocytes. More than a decade of development has recently resulted in the first approvals for this class of drugs. While substantial effort has been made to improve nucleic acid modification patterns for better payload stability and efficacy, relatively little attention has been given to the GalNAc targeting ligand. In addition, the lack of an intrinsic endosomal release mechanism has limited potency. Here, we report a stepwise analysis of the structure activity relationships (SAR) of the components comprising these targeting ligands. We show that there is relatively little difference in biological performance be"],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pagination":["2910-2919"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC8531135"],"repository":["biostudies-literature"],"pubmed_title":["Ligand conjugate SAR and enhanced delivery in NHP."],"pmcid":["PMC8531135"],"pubmed_authors":["Lee ACH","Heyes J","Majeski S","Ye X","Fraser D","McClintock K","Martin AD","Jarosz A","Thi EP","Wood MC","Judge A","Holland RJ","Palmer L","Lam K"],"additional_accession":[]},"is_claimable":false,"name":"Ligand conjugate SAR and enhanced delivery in NHP.","description":"N-Acetylgalactosamine (GalNAc) conjugated short interfering RNAs (siRNAs) are a leading RNA interference (RNAi) platform allowing targeted inhibition of disease-causing genes in hepatocytes. More than a decade of development has recently resulted in the first approvals for this class of drugs. While substantial effort has been made to improve nucleic acid modification patterns for better payload stability and efficacy, relatively little attention has been given to the GalNAc targeting ligand. In addition, the lack of an intrinsic endosomal release mechanism has limited potency. Here, we report a stepwise analysis of the structure activity relationships (SAR) of the components comprising these targeting ligands. We show that there is relatively little difference in biological performance be","dates":{"release":"2021-01-01T00:00:00Z","publication":"2021 Oct","modification":"2025-04-26T15:38:22.686Z","creation":"2025-04-06T14:57:01.159Z"},"accession":"S-EPMC8531135","cross_references":{"pubmed":["34091052"],"doi":["10.1016/j.ymthe.2021.06.002"]}}