Carelli · Progress in retinal and eye research 2004 · narrative review · n=?

Mitochondrial dysfunction as a cause of optic neuropathies.

Cited 842 times in the scientific literature.

Level 5 - mechanism / opinion, no new human data

Narrative review of mechanisms, genetics, and clinical phenotypes without systematic review methodology.

PubMed 14766317 · doi:10.1016/j.preteyeres.2003.10.003 · record verified 2026-08-29

What was done

This is a narrative review synthesizing anatomical, physiological, and genetic evidence on the role of mitochondrial dysfunction in optic neuropathies. The review describes mitochondrial distribution and axonal transport in retinal ganglion cells and discusses primary mitochondrial DNA disorders (such as Leber hereditary optic neuropathy [LHON]), nuclear gene defects (such as OPA1 mutations in dominant optic atrophy), toxic and nutritional optic neuropathies, and normal tension glaucoma, alongside experimental gene therapy approaches.

What was found

The abstract reports no quantitative data or effect estimates. It describes an asymmetric distribution of mitochondria in retinal ganglion cell axons, with high abundance in the unmyelinated prelaminar portion that sharply decreases in the retrobulbar myelinated tract. It outlines how diverse optic neuropathies (LHON, dominant optic atrophy, toxic/nutritional neuropathies, and potentially normal tension glaucoma) share a convergent pathomechanism involving impaired mitochondrial biogenesis, transport, or respiratory chain function. It also notes experimental attempts to correct LHON-related complex I dysfunction through allotopic nuclear expression of recoded mitochondrial genes.

Why it matters

The paper provides a unifying mechanistic framework linking disparate genetic and acquired optic neuropathies to the unique metabolic vulnerability and axonal distribution of mitochondria in retinal ganglion cells.

Limits

This is an unsystematic narrative review with no defined search protocol, study selection criteria, or quantitative synthesis. The abstract presents no original experimental numbers, clinical trial data, sample sizes, or human therapeutic outcome metrics.

Cited by