The beauty of symmetry: siRNA phosphorodithioate modifications reduce stereocomplexity, ease analysis, and can improve in vivo potency

Phosphorothioates (PSs) can be essential in stabilizing therapeutic oligonucleotides against enzymatic degradation. However, unless synthesis is performed with stereodefined amidites, each PS introduces a chemically undefined stereocenter, resulting in 2n unique molecules in the final product and af...

書誌詳細
出版年:	Molecular Therapy: Nucleic Acids
主要な著者:	Sophie Schöllkopf, Stefan Rathjen, Micaela Graglia, Nina Was, Eliot Morrison, Adrien Weingärtner, Lucas Bethge, Judith Hauptmann, Marie Wikström Lindholm
フォーマット:	論文
言語:	英語
出版事項:	Elsevier 2024-12-01
主題:	MT: Oligonucleotides: Therapies and Applications siRNA conjugate GalNAc phosphorothioate phosphorodithioate stereopure
オンライン･アクセス:	http://www.sciencedirect.com/science/article/pii/S2162253124002233

類似資料

Synthesis of 5′-GalNAc-Conjugated Oligonucleotides: A Comparison of Solid and Solution-Phase Conjugation Strategies
著者:: Isaiah Cedillo, 等
出版事項: (2017-08-01)

A modular dual-siRNA delivery platform based on GalNAc and click chemistry for multigene targeting
著者:: Xingliang Zhu, 等
出版事項: (2025-11-01)

Targeting Liver <i>Xor</i> by GalNAc-siRNA Is an Effective Strategy for Hyperuricemia Therapy
著者:: Huiyan Sun, 等
出版事項: (2024-07-01)

A Novel Pot-Economy Approach to the Synthesis of Triantennary GalNAc-Oligonucleotide
著者:: Artem Evgenievich Gusev, 等
出版事項: (2024-12-01)

Two Birds with One Stone: A Novel Dithiomaleimide-Based GalNAc-siRNA Conjugate Enabling Good siRNA Delivery and Traceability
著者:: Sudong Kong, 等
出版事項: (2023-10-01)

Therapeutic siRNA targeting PLIN2 ameliorates steatosis, inflammation, and fibrosis in steatotic liver disease models
著者:: Yao Wang, 等
出版事項: (2024-10-01)

Whole-Body Physiologically Based Pharmacokinetic Modeling of GalNAc-Conjugated siRNAs
著者:: Emilie Langeskov Salim, 等
出版事項: (2025-01-01)

Highly Potent GalNAc-Conjugated Tiny LNA Anti-miRNA-122 Antisense Oligonucleotides
著者:: Tsuyoshi Yamamoto, 等
出版事項: (2021-05-01)

The Gal/GalNac lectin as a possible acetylcholine receptor in Entamoeba histolytica
著者:: Marisol Pacheco-Sánchez, 等
出版事項: (2023-05-01)

Involvement of Fusobacterium nucleatum in malignancies except for colorectal cancer: A literature review
著者:: Zhixing He, 等
出版事項: (2022-08-01)

O‐glycosylation in viruses: A sweet tango
著者:: Annan Ming, 等
出版事項: (2024-03-01)

Whole-Body Physiologically Based Pharmacokinetic–Pharmacodynamic Modeling for Interspecies Translation and Mechanistic Characterization of Plasma and Tissue Disposition of GalNAc-siRNAs
著者:: Emilie Langeskov Salim, 等
出版事項: (2025-09-01)

Mapping the O‐glycoproteome using site‐specific extraction of O‐linked glycopeptides (EXoO)
著者:: Weiming Yang, 等
出版事項: (2018-11-01)

Protective Effects of GalNac-Modified Red Blood Cell-Derived Extracellular Vesicles Against Liver Diseases
著者:: Huang X, 等
出版事項: (2025-07-01)

Identification and characterization of a carbohydrate recognition domain-like region in Entamoeba histolytica Gal/GalNAc lectin intermediate subunit
著者:: Hongze Zhang, 等
出版事項: (2024-11-01)

<i>Entamoeba histolytica</i> Trophozoites Interact with the c-Met Receptor at the Surface of Liver Origin Cells through the Gal/GalNAc Amoebic Lectin
著者:: Jesus Pérez-Hernández, 等
出版事項: (2021-09-01)

Quantitative Proteomics Reveals Metabolic Reprogramming in Host Cells Induced by Trophozoites and Intermediate Subunit of Gal/GalNAc Lectins from Entamoeba histolytica
著者:: Yanqing Zhao, 等
出版事項: (2022-04-01)

Polypeptide <i>N</i>-acetylgalactosaminyltransferase-Associated Phenotypes in Mammals
著者:: Kentaro Kato, 等
出版事項: (2021-09-01)

Amniotic fluid glycoproteins as potential ligands for macrophage galactose-type C-type lectin and their possible implications for immunoregulation during pregnancy
著者:: Justyna Szczykutowicz, 等
出版事項: (2025-09-01)

Evaluation on Elongation Factor 1 Alpha of <i>Entamoeba histolytica</i> Interaction with the Intermediate Subunit of the Gal/GalNAc Lectin and Actin in Phagocytosis
著者:: Hang Zhou, 等
出版事項: (2020-08-01)

Investigation of the Protective Effect for GcMAF by a Glycosidase Inhibitor and the Glycan Structure of Gc Protein
著者:: Yoshimi Kanie, 等
出版事項: (2023-02-01)

GalNAc-T14 may Contribute to Production of Galactose-Deficient Immunoglobulin A1, the Main Autoantigen in IgA Nephropathy
著者:: Jana Jemelkova, 等
出版事項: (2023-05-01)

Functional Expression and Characterization of the Recombinant N-Acetyl-Glucosamine/N-Acetyl-Galactosamine-Specific Marine Algal Lectin BPL3
著者:: Hyun-Ju Hwang, 等
出版事項: (2018-01-01)

Analysis of the pharmacokinetics and efficacy of RBD1016 – A GalNAc-siRNA targeting Hepatitis B Virus X gene using semi-mechanistic PK/PD model
著者:: Qian Li, 等
出版事項: (2024-06-01)

Multi-omics insights into the metabolic reprogramming of host cells triggered by Entamoeba histolytica Gal/GalNAc lectin intermediate subunit
著者:: Yanqing Zhao, 等
出版事項: (2025-10-01)

New insights in amino sugar metabolism by the gut microbiome
著者:: Wenqiang Shen, 等
出版事項: (2025-12-01)

Liver-specific inactivation of Cideb improves metabolic profiles and ameliorates steatohepatitis and fibrosis in animal models for MASH
著者:: Jianhua Zhang, 等
出版事項: (2025-04-01)

Characterization of Human Recombinant β1,4-GalNAc-Transferase B4GALNT1 and Inhibition by Selected Compounds
著者:: Iram Abidi, 等
出版事項: (2025-09-01)

Enhancing cell-mediated immunity through dendritic cell activation: the role of Tri-GalNAc-modified PLGA-PEG nanoparticles encapsulating SR717
著者:: Yang Gong, 等
出版事項: (2024-12-01)

Corrigendum: Enhancing cell-mediated immunity through dendritic cell activation: the role of Tri-GalNAc-modified PLGA-PEG nanoparticles encapsulating SR717
著者:: Yang Gong, 等
出版事項: (2025-04-01)

The utilization of N-acetylgalactosamine and its effect on the metabolism of amino acids in Erysipelotrichaceae strain
著者:: Mengqing Zhou, 等
出版事項: (2024-10-01)

Development of glycan-targeted nanoparticles as a novel therapeutic opportunity for gastric cancer treatment
著者:: Sofia Nascimento dos Santos, 等
出版事項: (2023-03-01)

EhVps35, a retromer component, is involved in the recycling of the EhADH and Gal/GalNac virulent proteins of Entamoeba histolytica
著者:: Joselin Díaz-Valdez, 等
出版事項: (2024-03-01)

The macrophage polarization in Entamoeba histolytica infection modulation by the C fragment of the intermediate subunit of Gal/GalNAc-inhibitable lectin
著者:: Dai Dong, 等
出版事項: (2024-07-01)

In-silico prediction and modeling of the Entamoeba histolytica proteins: Serine-rich Entamoeba histolytica protein and 29 kDa Cysteine-rich protease
著者:: Kumar Manochitra, 等
出版事項: (2017-06-01)

RAB18 is a key regulator of GalNAc-conjugated siRNA-induced silencing in Hep3B cells
著者:: Jiamiao Lu, 等
出版事項: (2022-06-01)

Sialyl-Tn in Cancer: (How) Did We Miss the Target?
著者:: Philippe Delannoy, 等
出版事項: (2012-10-01)

Analysis of cancer-associated glycosyltransferases reveals novel targets of non-small cell lung cancer pathogenesis
著者:: Chang Liu, 等
出版事項: (2025-07-01)

Phosphorothioate DNA Mediated Sequence-Insensitive Etching and Ripening of Silver Nanoparticles
著者:: Shengqiang Hu, 等
出版事項: (2019-04-01)

Preclinical evaluation of WVE-004, aninvestigational stereopure oligonucleotide forthe treatment of C9orf72-associated ALS or FTD
著者:: Yuanjing Liu, 等
出版事項: (2022-06-01)