Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

OPA1 gene therapy prevents retinal ganglion cell loss in a Dominant Optic Atrophy mouse model.

Scientific reports - 6 Feb 2018

Sarzi Emmanuelle, Seveno Marie, Piro-Mégy Camille, Elzière Lucie, Quilès Mélanie, Péquignot Marie, Müller Agnès, Hamel Christian P, Lenaers Guy, Delettre Cécile

Abstract excerpt

Dominant optic atrophy (DOA) is a rare progressive and irreversible blinding disease which is one of the most frequent forms of hereditary optic neuropathy. DOA is mainly caused by dominant mutation in the OPA1 gene encoding a large mitochondrial GTPase with crucial roles in membrane dynamics and cell survival. Hereditary optic neuropathies are commonly characterized by the degeneration of retinal ganglion cells,...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Cell Death
Share to Cytomegalovirus

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Dependovirus

Share to GTP Phosphohydrolases

Share to Intravitreal Injections

Share to Optic Atrophy, Autosomal Dominant

Share to Optic Nerve

Share to Retinal Ganglion Cells

Source

Open in PubMed Open full text

Identifiers

PMID: 29410463
DOI: 10.1038/s41598-018-20838-8
PMCID: PMC5802757

Genes

OPA1

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.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

OPA1 gene therapy prevents retinal ganglion cell loss in a Dominant Optic Atrophy mouse model.PMID 29410463Read ↗

The human OPA1delTTAG mutation induces premature age-related systemic neurodegeneration in mouse.Semantic proximity Read ↗

Targeting the OPA1 pathway in Autosomal Dominant Optic Atrophy (ADOA): 25 years from gene discovery to therapeutic strategy.Semantic proximity Read ↗

Dominant optic atrophy: updates on the pathophysiology and clinical manifestations of the optic atrophy 1 mutation.Semantic proximity Read ↗

OPA1 Dominant Optic Atrophy: Pathogenesis and Therapeutic Targets.Semantic proximity Read ↗

A splice site mutation in the murine Opa1 gene features pathology of autosomal dominant optic atrophy.Semantic proximity Read ↗

[A murine model of transitory optic neuropathy based on small interference RNA-induced OPA1 silencing in vivo (gene mutation associated with Kjer's disease)].Semantic proximity Read ↗

Adeno-associated virus vectors for retinal gene therapy in basic research and clinical studiesSemantic proximity Read ↗

Mitochondrial dysfunction in an Opa1(Q285STOP) mouse model of dominant optic atrophy results from Opa1 haploinsufficiency.Semantic proximity Read ↗

OPA1: How much do we know to approach therapy?Semantic proximity Read ↗

Creation of an Isogenic Human iPSC-Based RGC Model of Dominant Optic Atrophy Harboring the Pathogenic Variant c.1861C>T (p.Gln621Ter) in the OPA1 Gene.Semantic proximity Read ↗

[Not only optic neuropathy: new molecular and clinical aspects of OPA1 gene mutations].Semantic proximity Read ↗

The concept of gene therapy for glaucomaSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.