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Biomedicines 2018, 6(1), 2; https://doi.org/10.3390/biomedicines6010002

The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders

1
INSERM U1179, UFR des Sciences de la Santé, University of Versailles St-Quentin, 78180 Montigny le Bretonneux, France
2
SQY Therapeutics, University of Versailles St-Quentin, 78180 Montigny le Bretonneux, France
These authors contributed equally to this work.
*
Author to whom correspondence should be addressed.
Received: 30 November 2017 / Revised: 13 December 2017 / Accepted: 19 December 2017 / Published: 22 December 2017
(This article belongs to the Special Issue Antisense Therapy)
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Abstract

Antisense Oligonucleotides (ASOs) represent very attractive therapeutic compounds for the treatment of numerous diseases. The antisense field has remarkably progressed over the last few years with the approval of the first antisense drugs and with promising developments of more potent and nuclease resistant chemistries. Despite these recent clinical successes and advances in chemistry and design, effective delivery of ASOs to their target tissues remains a major issue. This review will describe the latest advances obtained with the tricyclo-DNA (tcDNA) chemistry which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration. We will examine the variety of therapeutic approaches using both fully modified tcDNA-ASOs and gapmers, including splice switching applications, correction of aberrant splicing, steric blocking strategies and targeted gene knock-down mediated by RNase H recruitment. We will then discuss the merits and potential liabilities of the tcDNA chemistry in the context of ASO drug development. View Full-Text
Keywords: antisense oligonucleotides; tricyclo-DNA; gapmers; splice-switching; exon-skipping; exon-re-inclusion; delivery antisense oligonucleotides; tricyclo-DNA; gapmers; splice-switching; exon-skipping; exon-re-inclusion; delivery
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).
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Aupy, P.; Echevarría, L.; Relizani, K.; Goyenvalle, A. The Use of Tricyclo-DNA Oligomers for the Treatment of Genetic Disorders. Biomedicines 2018, 6, 2.

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