OPA1 adenovirus

OPA1 adenovirus refers to an adenoviral vector engineered to deliver and express the human OPA1 gene, most commonly for gene therapy applications in disorders associated with mitochondrial dysfunction, particularly dominant optic atrophy (DOA). OPA1 is a mitochondrial inner membrane protein crucial for mitochondrial fusion, cristae structure, and cellular survival. Delivery of OPA1 via adenovirus has been shown in preclinical studies to protect retinal ganglion cells and restore mitochondrial function in models of DOA and other mitochondrial disorders. The adenovirus serves as a vehicle to introduce functional OPA1 cDNA into target cells, thereby addressing OPA1 haploinsufficiency and improving mitochondrial bioenergetics. The approach is experimental and typically administered via local routes (such as intravitreal injection) for ophthalmic indications.