Mitochondria are organelles with vital functions in almost all eukaryotic cells. Often
described as the cellular 'powerhouses' due to their essential role in aerobic oxidative …

Mitochondrially generated reactive oxygen species are involved in a myriad of signaling and
damaging pathways in different tissues. In addition, mitochondria are an important target of …

Leber hereditary optic neuropathy (LHON) is one of the most common inherited optic
neuropathies causing bilateral central vision loss. The disorder results from point mutations …

Blindness, although not life threatening, is a debilitating disorder for which few, if any
treatments exist. Ocular gene therapies have the potential to profoundly improve the quality …

Mitochondrial diseases frequently manifest neuro-ophthalmologic symptoms and signs.
Because of the predilection of mitochondrial disorders to involve the optic nerves …

Inherited retinopathies (IR) are common untreatable blinding conditions. Most of them are
inherited as monogenic disorders, due to mutations in genes expressed in retinal …

Bilateral visual loss secondary to inherited optic neuropathies is an important cause of
registrable blindness among children and young adults. The two prototypal disorders seen …

Using mouse optic nerve (ON) crush as a CNS injury model, we and others have found that
activation of the mammalian target of rapamycin complex 1 (mTORC1) in mature retinal …

To introduce DNA into mitochondria efficiently, we fused adenoassociated virus capsid VP2
with a mitochondrial targeting sequence to carry the mitochondrial gene encoding the …

Mitochondrial diseases (MD) are a heterogeneous group of multisystem disorders involving
metabolic errors. MD are characterized by extremely heterogeneous symptoms, ranging …