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 …

Adeno-associated viral vectors (AAVs) are increasingly useful preclinical tools in
neuroscience research studies for interrogating cellular and neurocircuit functions and …

Inherited retinal degenerations are caused by mutations in> 250 genes that affect
photoreceptor cells or the retinal pigment epithelium and result in vision loss. For autosomal …

Vectors based on adeno-associated virus serotype 2 (AAV2) have been used extensively in
many gene-delivery applications, including several successful clinical trials for one type of …

Retinitis pigmentosa (RP) and age-related macular degeneration (AMD) are degenerative
blinding diseases caused by the death of rods and cones, leaving the remainder of 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 …

Abstract Streptococcus pyogenes (Spy) Cas9 has potential as a component of gene
therapeutics for incurable diseases. One of its limitations is its large size, which impedes its …

Development of viral vectors capable of transducing photoreceptors by less invasive
methods than subretinal injection would provide a major advancement in retinal gene …

Vectors derived from adeno-associated virus (AAV) are currently the most promising
vehicles for therapeutic gene delivery to the retina. Recently, subretinal administration of …

Positive clinical outcomes in adeno-associated virus (AAV)-mediated retinal gene therapy
have often been attributed to the low immunogenicity of AAVs and immune privilege of the …