Adeno-associated virus (AAV) has proven an effective gene delivery vehicle for the treatment
of retinal disease. Ongoing clinical trials using a serotype 2 AAV vector to express RPE65 …

Geographic atrophy (GA), an untreatable advanced form of age-related macular degeneration,
results from retinal pigmented epithelium (RPE) cell degeneration. Here we show that the …

Recombinant adeno-associated viral (rAAV) vectors based on serotype 2 are currently being
evaluated most extensively in animals and human clinical trials. rAAV vectors constructed …

Alu RNA accumulation due to DICER1 deficiency in the retinal pigmented epithelium (RPE)
is implicated in geographic atrophy (GA), an advanced form of age-related macular …

The successful restoration of visual function with recombinant adeno-associated virus (rAAV)-mediated
gene replacement therapy in animals and humans with an inherited disease of …

Hereditary retinal blindness is caused by mutations in genes expressed in photoreceptors
or retinal pigment epithelium. Gene therapy in mouse and dog models of a primary retinal …

Retinal pigment epithelium-specific protein 65 kDa (RPE65) is a protein responsible for
isomerization of all-trans-retinaldehyde to its photoactive 11-cis-retinaldehyde and is essential …

IL-10 is a pluripotent cytokine that plays a pivotal role in the regulation of immune and
inflammatory responses. Whereas short-term administration of IL-10 has shown benefit in acute …

Usher syndrome 1B (USH1B) is a severe, autosomal recessive, deaf–blind disorder caused
by mutations in myosin 7A (MYO7A). Patients are born profoundly deaf and exhibit …

Achromatopsia is a rare autosomal recessive disorder which shows color blindness,
severely impaired visual acuity, and extreme sensitivity to bright light. Mutations in the alpha …