Recombinant adeno-associated virus vectors (rAAV) are being explored as gene delivery
vehicles for the treatment of various inherited and acquired disorders. rAAVs are attractive …

Age-related macular degeneration (AMD) is one of the leading causes of irreversible
blindness in the developed world. Antivascular endothelial growth factor therapy has …

RP2 mutations cause a severe form of X-linked retinitis pigmentosa (XLRP). The mechanism
of RP2-associated retinal degeneration in humans is unclear, and animal models of RP2 …

Targeting genes to specific neuronal or glial cell types is valuable for both understanding
and repairing brain circuits. Adeno-associated viruses (AAVs) are frequently used for gene …

Retinal gene therapy with adeno‐associated viral (AAV) vectors is safe and effective in
humans. However, AAV's limited cargo capacity prevents its application to therapies of …

The use of adeno-associated viral (AAV) vectors for gene therapy treatments of inherited
disorders has accelerated over the past decade with multiple clinical trials ongoing in …

Gene therapy using adeno-associated viral (AAV) vectors currently represents the most
promising approach for the treatment of many inherited retinal diseases (IRDs), given AAV's …

Abstract Adeno-associated virus (AAV)-mediated gene therapy has evolved from bench to
bedside, and now is the therapy of choice for certain inherited diseases. However, the small …

Retinal gene therapy has advanced considerably in the past three decades. Initial efforts
have been devoted to comprehensively explore and optimize the transduction abilities of …

Adeno-associated virus (AAV) vectors are the most widely used vehicle systems for
neuronal gene transfer. This popularity is based on the non-pathogenic nature of AAVs and …