Mitochondria are subject to unique genetic control by both nuclear DNA and their own
genome, mitochondrial DNA (mtDNA), of which each mitochondrion contains multiple …

The past decade has seen an explosion of research directed toward better understanding of
the mechanisms of mesenchymal stem/stromal cell (MSC) function during rescue and repair …

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-
associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential …

Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders
that result from dysfunction of the mitochondrial oxidative phosphorylation due to molecular …

Purpose Leber hereditary optic neuropathy (LHON) is a disorder characterized by severe
and rapidly progressive visual loss when caused by a mutation in the mitochondrial gene …

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 …

Background: Targeting of drugs to the subcellular compartments represents one of the
modern trends in molecular pharmacology. The approach for targeting mitochondria was …

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 …

Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …

Members of the virus family Narnaviridae contain the simplest genomes of any RNA virus,
ranging from 2.3 to 3.6 kb and encoding only a single polypeptide that has an RNA …