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Modular and automated synthesis of oligonucleotide-small molecule conjugates for cathepsin B mediated traceless release of payloads

Modular and automated synthesis of oligonucleotide-small molecule conjugates for cathepsin B mediated traceless release of payloads
Modular and automated synthesis of oligonucleotide-small molecule conjugates for cathepsin B mediated traceless release of payloads
The reversible attachment of small molecules to oligonucleotides provides versatile tools for the development of improved oligonucleotide therapeutics. However, cleavable linkers in the oligonucleotide field are scarce, particularly with respect to the requirement for traceless release of the payload in vivo. Herein, we describe a cathepsin B-cleavable dipeptide phosphoramidite, Val-Ala(NB) for the automated synthesis of oligonucleotide-small molecule conjugates. Val-Ala(NB) was protected by a photolabile 2-nitrobenzyl group to improve the stability of the peptide linker during DNA synthesis. Intracellular cathepsin B digests the dipeptide efficiently, releasing the payload-phosphate which is converted to the free payload by endogenous phosphatase enzymes. With the advantages of modular synthesis and stimuli-responsive drug release, we believe Val-Ala(NB) will be a potentially valuable cleavable linker for use in oligonucleotide-drug conjugates.
2633-0679
738-744
Jin, Cheng
626b74f9-e46d-48f0-879a-d58fdc0e0f1e
Li, Siqi
e41d2d81-2399-461a-85f2-7c5a81cbcd32
Vallis, Katherine
d20e2712-1b86-4201-97e5-0a4b1e16d107
El-Sagheer, Afaf
05b8295a-64ad-4fdf-ad57-c34934a46c04
Brown, Tom
a64aae36-bb30-42df-88a2-11be394e8c89
Jin, Cheng
626b74f9-e46d-48f0-879a-d58fdc0e0f1e
Li, Siqi
e41d2d81-2399-461a-85f2-7c5a81cbcd32
Vallis, Katherine
d20e2712-1b86-4201-97e5-0a4b1e16d107
El-Sagheer, Afaf
05b8295a-64ad-4fdf-ad57-c34934a46c04
Brown, Tom
a64aae36-bb30-42df-88a2-11be394e8c89

Jin, Cheng, Li, Siqi, Vallis, Katherine, El-Sagheer, Afaf and Brown, Tom (2024) Modular and automated synthesis of oligonucleotide-small molecule conjugates for cathepsin B mediated traceless release of payloads. RSC Chemical Biology, 5 (8), 738-744. (doi:10.1039/D4CB00112E).

Record type: Article

Abstract

The reversible attachment of small molecules to oligonucleotides provides versatile tools for the development of improved oligonucleotide therapeutics. However, cleavable linkers in the oligonucleotide field are scarce, particularly with respect to the requirement for traceless release of the payload in vivo. Herein, we describe a cathepsin B-cleavable dipeptide phosphoramidite, Val-Ala(NB) for the automated synthesis of oligonucleotide-small molecule conjugates. Val-Ala(NB) was protected by a photolabile 2-nitrobenzyl group to improve the stability of the peptide linker during DNA synthesis. Intracellular cathepsin B digests the dipeptide efficiently, releasing the payload-phosphate which is converted to the free payload by endogenous phosphatase enzymes. With the advantages of modular synthesis and stimuli-responsive drug release, we believe Val-Ala(NB) will be a potentially valuable cleavable linker for use in oligonucleotide-drug conjugates.

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d4cb00112e - Accepted Manuscript
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Accepted/In Press date: 28 May 2024
e-pub ahead of print date: 29 May 2024
Published date: 29 May 2024
Additional Information: Publisher Copyright: © 2024 RSC

Identifiers

Local EPrints ID: 490870
URI: http://eprints.soton.ac.uk/id/eprint/490870
ISSN: 2633-0679
PURE UUID: 2ebcd182-837f-4b74-bc05-d1f6a05edccf
ORCID for Afaf El-Sagheer: ORCID iD orcid.org/0000-0001-8706-1292

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Date deposited: 07 Jun 2024 16:39
Last modified: 12 Dec 2024 02:40

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Contributors

Author: Cheng Jin
Author: Siqi Li
Author: Katherine Vallis
Author: Afaf El-Sagheer ORCID iD
Author: Tom Brown

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