Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …

Gene therapies offer promising therapeutic alternatives for many disorders that currently lack
efficient treatment options. Due to their chemical nature and physico-chemical properties …

Adeno-associated viral vectors (AAVs) have become popular for gene therapy, given their
many advantages, including their reduced inflammatory profile compared with that of other …

Achromatopsia (ACHM), also known as rod monochromatism or total color blindness, is an
autosomal recessively inherited retinal disorder that affects the cones of the retina, the type …

The recent approval of voretigene neparvovec (Luxturna®) for patients with biallelic RPE65
mutation-associated inherited retinal dystrophy with viable retinal cells represents an …

Studies utilizing large animal models of inherited retinal degeneration (IRD) have proven
important in not only the development of translational therapeutic approaches, but also in …

The first step in vision is the absorption of photons by the photopigments in cone and rod
photoreceptors. After initial amplification within the phototransduction cascade the signal is …

Inherited retinal degenerations are a leading and untreatbale cause of blindness, and as
such they are targets for gene therapy. Numerous gene therapy treatments have progressed …

Gene therapy is a promising approach in the treatment of inherited and common complex
disorders of the retina. Preclinical and clinical studies have validated the use of adeno …

The visual process begins with the absorption of photons by photopigments of cone and rod
photoreceptors in the retina. In this process, the signal is first amplified by a cyclic guanosine …