Dynamin-like GTPase OPA1, mitochondrial (OPA1)

OPA1 is a mitochondrial inner membrane GTPase essential for mitochondrial inner membrane fusion, maintenance of cristae structure, and the integrity of mitochondrial DNA. It exists in multiple splice isoforms, localizes to the inner mitochondrial membrane, and is required for normal mitochondrial dynamics and energy production. Dysfunction or mutation of OPA1 leads to mitochondrial disorganization and increased susceptibility to apoptosis, contributing to dominantly inherited optic neuropathy (DOA), various neurological syndromes, and other mitochondrial diseases. Therapeutic targeting of OPA1 mRNA with antisense oligonucleotides is under investigation as a strategy to increase functional OPA1 protein in disorders such as dominant optic atrophy[1][2][3][4][5][6][8].

Other names

Optic atrophy protein 1OPA1 mitochondrial dynamin-like GTPaseDynamin-like 120 kDa protein, Kjer type

Splice-switching antisense oligonucleotides decrease inclusion of poison exons in OPA1 mRNA, increasing production of functional OPA1 protein