Gene delivery vectors based on adeno-associated viruses (AAV) have exhibited promise in
both preclinical disease models and human clinical trials for numerous disease targets …

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 …

Viral vectors can be utilised to deliver therapeutic genes to diseased cells. Adeno‐
associated virus (AAV) is a commonly used viral vector that is favoured for its ability to infect …

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 …

Background Adeno‐associated virus serotype 2 (AAV2) vectors show considerable promise
for ocular gene transfer. However, one potential barrier to efficacious long‐term therapy is …

The seroprevalence of neutralizing antibodies (NAbs) to adeno-associated viral (AAV)
vector capsids may preclude a percentage of the population from receiving gene therapy …

The eye has been at the forefront of translational gene therapy largely owing to suitable
disease targets, anatomic accessibility, and well-studied immunologic privilege. These …

Purpose Safety and efficiency are critical for successful gene therapy. Adeno-associated
viral (AAV) vectors are commonly used for gene transfer in both human and animal studies …

There has been significant progress in the last few years in demonstrating the utility of
recombinant viral vectors in treating a variety of ocular diseases. The field has moved …

SUMMARY AAV‐based gene transfer protocols have shown remarkable success when
directed to immune‐privileged sites such as for retinal disorders like Lebers congenital …