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Article

Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases.

Expert opinion on investigational drugs - 1 Oct 2020

Xue Kanmin, MacLaren Robert E

Abstract excerpt

INTRODUCTION: Antisense oligonucleotides (ASOs) represent a class of drugs which can be rationally designed to complement the coding or non-coding regions of target RNA transcripts. They could modulate pre-messenger RNA splicing, induce mRNA knockdown, or block translation of disease-causing genes, thereby slowing disease progression. The pharmacokinetics of intravitreal delivery may enable ASOs to be effective...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 32741234
DOI: 10.1080/13543784.2020.1804853

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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