AAV-NDI1

AAV-NDI1 is an investigational gene therapy comprised of an adeno-associated viral (AAV) vector delivering the *NDI1* gene, a nuclear-encoded yeast gene for NADH-quinone oxidoreductase that replaces mitochondrial complex I function. In mammalian and human models of optic neuropathies, including glaucoma and Leber hereditary optic neuropathy (LHON), AAV-NDI1 improves mitochondrial function, increases oxygen consumption and ATP production, and reduces oxidative stress, providing neuroprotection to retinal ganglion cells and preserving retinal function. Preclinical efficacy has been demonstrated using intravitreal administration in animal models of complex I deficiency.[1][2][3][5][6]

Other names

adeno-associated virus NDI1AAV-eNdi1AAV-eNdi-1AAV-eNdi 1ophNdi1ophNdi-1ophNdi 1