Article
Targeting the OPA1 pathway in Autosomal Dominant Optic Atrophy (ADOA): 25 years from gene discovery to therapeutic strategy.
Expert opinion on therapeutic targets - 1 May 2026
Alavi Marcel V
Abstract excerpt
INTRODUCTION: Autosomal Dominant Optic Atrophy (ADOA) is a rare hereditary optic neuropathy primarily caused by OPA1 mutations. Retinal ganglion cell (RGC) loss results in variable visual impairments, occasionally accompanied by extra-ocular manifestations. ADOA also involves a developmental component consistent with OPA1's essential role in mitochondrial fusion, cristae organization, and quality control. As...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
