Properties of Parallel Tetramolecular G-Quadruplex Carrying

G-quadruplex N-acetylgalactosamine antisense asialoglycoprotein receptor gapmers luciferase gene oligonucleotide conjugates

Journal

Molecules (Basel, Switzerland)
ISSN: 1420-3049
Titre abrégé: Molecules
Pays: Switzerland
ID NLM: 100964009

Informations de publication

Date de publication:
20 Jun 2022
Historique:
received: 13 05 2022
revised: 08 06 2022
accepted: 17 06 2022
entrez: 24 6 2022
pubmed: 25 6 2022
medline: 28 6 2022
Statut: epublish

Résumé

The development of oligonucleotide conjugates for in vivo targeting is one of the most exciting areas for oligonucleotide therapeutics. A major breakthrough in this field was the development of multifunctional GalNAc-oligonucleotides with high affinity to asialoglycoprotein receptors (ASGPR) that directed therapeutic oligonucleotides to hepatocytes. In the present study, we explore the use of G-rich sequences functionalized with one unit of GalNAc at the 3'-end for the formation of tetrameric GalNAc nanostructures upon formation of a parallel G-quadruplex. These compounds are expected to facilitate the synthetic protocols by providing the multifunctionality needed for the binding to ASGPR. To this end, several G-rich oligonucleotides carrying a TGGGGGGT sequence at the 3'-end functionalized with one molecule of

Identifiants

pubmed: 35745067
pii: molecules27123944
doi: 10.3390/molecules27123944
pmc: PMC9228010
pii:
doi:

Substances chimiques

Asialoglycoprotein Receptor 0
Oligonucleotides 0
Acetylgalactosamine KM15WK8O5T

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : Biomedical Research Networking Center in Bioengineering, Biomaterials and Nanomedicine
ID : cb06/01/0019
Organisme : spanish ministerio de ciencia e innovación
ID : CTQ2017-84415-R
Organisme : spanish ministerio de ciencia e innovación
ID : PID2020-118145RB-I00
Organisme : spanish ministerio de ciencia e innovación
ID : PID2019-107158GB-I100
Organisme : PIANO DI SOSTEGNO ALLA RICERCA 2020
ID : Linea 2 azione B (DEFENS)
Organisme : MICINN
ID : PRE2018-084056

Commentaires et corrections

Type : ErratumIn

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Auteurs

Anna Clua (A)

Institute for Advanced Chemistry of Catalonia (IQAC-CSIC), Jordi Girona 18-26, E-08034 Barcelona, Spain.
Networking Center on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), E-08034 Barcelona, Spain.

Santiago Grijalvo (S)

Institute for Advanced Chemistry of Catalonia (IQAC-CSIC), Jordi Girona 18-26, E-08034 Barcelona, Spain.
Networking Center on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), E-08034 Barcelona, Spain.

Namrata Erande (N)

Alnylam Pharmaceuticals, 300 Third Street, Cambridge, MA 02142, USA.

Swati Gupta (S)

Alnylam Pharmaceuticals, 300 Third Street, Cambridge, MA 02142, USA.

Kristina Yucius (K)

Alnylam Pharmaceuticals, 300 Third Street, Cambridge, MA 02142, USA.

Raimundo Gargallo (R)

Department of Chemical Engineering and Analytical Chemistry, University of Barcelona, Martí i Franquès 1-11, E-08028 Barcelona, Spain.

Stefania Mazzini (S)

DEFENS-Dipartimento Di Scienze per Gli Alimenti, la Nutrizione e l'Ambiente, Università degli Studi di Milano, Via Celoria, 2, 20133 Milan, Italy.

Muthiah Manoharan (M)

Alnylam Pharmaceuticals, 300 Third Street, Cambridge, MA 02142, USA.

Ramon Eritja (R)

Institute for Advanced Chemistry of Catalonia (IQAC-CSIC), Jordi Girona 18-26, E-08034 Barcelona, Spain.
Networking Center on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), E-08034 Barcelona, Spain.

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Classifications MeSH