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The OPA1 protein is a dynamin-related GTPase localized to the inner mitochondrial membrane. It is essential for maintaining normal mitochondrial morphology and function. OPA1 mediates the fusion of mitochondrial inner membranes and regulates cristae structure, which is crucial for efficient oxidative phosphorylation. It also helps maintain mitochondrial DNA integrity and participates in controlled cell death (apoptosis). Mutations in OPA1 cause autosomal dominant optic atrophy (DOA) and have been implicated in neurodegenerative diseases. Therapeutic approaches under investigation include antisense oligonucleotides designed to increase functional OPA1 protein levels for DOA treatment.
Antisense oligonucleotides designed to increase functional OPA1 protein levels.
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