Until recently, there was no approved treatment for a retinal degenerative disease.
Subretinal injection of a recombinant adeno-associated virus (AAV) delivering the normal …

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 …

Choroideremia is a rare, X-linked retinal degeneration resulting in progressive vision loss. A
randomized, masked, phase 3 clinical trial evaluated the safety and efficacy over 12 months …

The relationship between the neurosensory photoreceptors and the adjacent retinal pigment
epithelium (RPE) controls not only normal retinal function, but also the pathogenesis of …

The death of photoreceptor cells caused by retinal degenerative diseases often results in a
complete loss of retinal responses to light. We explore the feasibility of converting inner …

Demonstration of safe and stable reversal of blindness after a single unilateral subretinal
injection of a recombinant adeno-associated virus (AAV) carrying the RPE65 gene (AAV2 …

The short-and long-term effects of gene therapy using AAV-mediated RPE65 transfer to
canine retinal pigment epithelium were investigated in dogs affected with disease caused by …

Recombinant vectors based on adeno-associated virus (AAV) or human immunodeficiency
1 (lentivirus) are promising tools for long term in vivo gene delivery. Their design allows the …

Gene therapy is emerging as a therapeutic modality for treating disorders of the retina.
Photoreceptor cells are the primary cell type affected in many inherited diseases of retinal …

Nonsyndromic recessive retinal dystrophies cause severe visual impairment due to the
death of photoreceptor and retinal pigment epithelium cells. These diseases until recently …