O'Sullivan, Joseph, Munoz, Jose, Turnbull, Graeme, Sim, Neil, Penny, Stuart and Moschos, Sterghios (2022) Beyond GalNAc! Drug delivery systems comprising complex oligosaccharides for targeted use of nucleic acid therapeutics. RSC Advances, 12 (32). pp. 20432-20446. ISSN 2046-2069
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Abstract
Nucleic Acid Therapeutics (NATs) are establishing a leading role for the management and treatment of genetic diseases following FDA approval of nusinersen, patisiran, and givosiran in the last 5 years, the breakthrough of milasen, with more approvals undoubtedly on the way. Givosiran takes advantage of the known interaction between the hepatocyte specific asialoglycoprotein receptor (ASGPR) and N-acetyl galactosamine (GalNAc) ligands to deliver a therapeutic effect, underscoring the value of targeting moieties. In this review, we explore the history of GalNAc as a ligand, and the paradigm it has set for the delivery of NATs through precise targeting to the liver, overcoming common hindrances faced with this type of therapy. We describe various complex oligosaccharides (OSs) and ask what others could be used to target receptors for NAT delivery and the opportunities awaiting exploration of this chemical space.
| Item Type: | Article |
|---|---|
| Additional Information: | Funding information: The authors are grateful for the financial support of High Force Research in this research. |
| Subjects: | B900 Others in Subjects allied to Medicine C700 Molecular Biology, Biophysics and Biochemistry |
| Department: | Faculties > Health and Life Sciences > Applied Sciences |
| Depositing User: | Rachel Branson |
| Date Deposited: | 15 Jul 2022 08:12 |
| Last Modified: | 03 Aug 2022 11:15 |
| URI: | http://nrl.northumbria.ac.uk/id/eprint/49551 |
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