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Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells.

Nucleic acid therapeutics - 1 Oct 2024

Venkatesh Aditya, McKenty Taylor, Ali Syed, Sonntag Donna, Ravipaty Shobha, Cui Yanyan, Slate Deirdre, Lin Qian, Christiansen Anne, Jacobson Sarah, Kach Jacob, Lim Kian Huat, Srinivasan Vaishnavi, Zinshteyn Boris, Aznarez Isabel, Huryn Laryssa A, Li Zhiyu, Hufnagel Robert B, Liau Gene, Anderson Karen, Hoger Jeff

Abstract excerpt

Autosomal dominant optic atrophy (ADOA) is an inherited optic neuropathy most frequently associated with OPA1 mutations. Most variants result in haploinsufficiency, and patient cells express roughly half of the normal levels of OPA1 protein. OPA1 is a mitochondrial GTPase that is essential for normal mitochondrial function. We identified and characterized STK-002, an antisense oligonucleotide (ASO) designed to...

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Identifiers

PMID: 39264859
DOI: 10.1089/nat.2024.0022
PMCID: PMC11564677

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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