Article

Delivery of GalNAc-conjugated splice-switching ASOs to non-hepatic cells through ectopic expression of asialoglycoprotein receptor

2018-08-02

Abstract excerpt

Splice-switching antisense oligonucleotides (ASOs) are promising therapeutic tools to target various genetic diseases, including cancer. However, in vivo delivery of ASOs to orthotopic tumors in cancer mouse models or to certain target tissues remains challenging. A viable solution already in use is receptor-mediated uptake of ASOs via tissue-specific receptors. For example, the asialoglycoprotein receptor (ASGP-...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 1781028a-070f-5f59-a2d7-8a884fe000fb
DOI: 10.1101/382606

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Delivery of GalNAc-conjugated splice-switching ASOs to non-hepatic cells through ectopic expression of asialoglycoprotein receptorDOI 10.1101/382606Read

Preclinical Screening of Splice-Switching Antisense Oligonucleotides in PDAC OrganoidsSemantic proximity Read

Delivery is key: lessons learnt from developing splice‐switching antisense therapiesSemantic proximity Read

Advancing Liver Gene Therapy: Enhanced Transduction with GalNAc-Bioconjugated rAAV CapsidsSemantic proximity Read

RAB18 is a key regulator of GalNAc conjugated siRNA induced silencing in Hep3B cellsSemantic proximity Read

Splice-Switching Antisense Oligonucleotides as a Targeted Intrinsic Engineering Tool for Generating Armored Redirected T Cells.Semantic proximity Read

Targeting stroma and tumor, silencing galectin 1 treats orthotopic mouse hepatocellular carcinomaSemantic proximity Read

Whole-Body Physiologically-Based Pharmacokinetic Modeling of GalNAc conjugated siRNAsSemantic proximity Read

Antisense gapmers selectively suppress individual oncogenic p73 splice isoforms and inhibit tumor growth in vivoSemantic proximity Read

T Cells Engineered to Express a T-Cell Receptor Specific for Glypican-3 to Recognize and Kill Hepatoma Cells In Vitro and in MiceSemantic proximity Read

Nipah Virus Envelope-Pseudotyped Lentiviruses Efficiently Target ephrinB2-Positive Stem Cell Populations In Vitro and Bypass the Liver Sink When Administered In VivoSemantic proximity Read

Efficacy and safety of glycosphingolipid SSEA-4 targeting CAR-T cells in an ovarian carcinoma modelSemantic proximity Read

Suppression of porcine xenoantigen expression by dominant-negative effect of alpha-1,3-galactosyltransferase (alpha-1,3-GT) splicing variants.Semantic proximity Read