Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …

Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

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 …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

The field of gene therapy has made considerable progress over the past several years.
Adeno-associated virus (AAV) vectors have emerged as promising and attractive tools for in …

Immunogenicity has imposed a challenge to efficacy and safety evaluation of adeno-
associated virus (AAV) vector-based gene therapies. Mild to severe adverse events …

Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene
therapy and has been widely used in> 200 clinical trials globally. There are already several …

As adeno-associated virus (AAV)-based gene therapies are being increasingly approved for
use in humans, it is important that we understand vector–host interactions in detail. With the …

Recombinant adeno-associated virus (rAAV) is a clinically proven viral vector for delivery of
therapeutic genes to treat rare diseases. Improving rAAV manufacturing productivity and …