The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

The ability to elicit robust and long-term transgene expression in vivo together with minimal
immunogenicity and little to no toxicity are only a few features that make recombinant adeno …

Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Adeno-associated virus (AAV) has shown great promise as a gene therapy vector in multiple
aspects of preclinical and clinical applications. Many developments including new serotypes …

Recombinant adeno-associated viral (AAV) vectors have rapidly advanced to the forefront of
gene therapy in the past decade. The exponential progress of AAV-based vectors has been …

The adeno-associated virus (AAV) is a small, nonpathogenic parvovirus, which depends on
helper factors to replicate. Those helper factors can be provided by coinfecting helper …

The adeno-associated virus (AAV) is one of the most promising viral vectors for human gene
therapy. As with any potential therapeutic system, a thorough understanding of it at the in …

Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α1-
antitrypsin (AAT) deficiency. In our prior trial, an rAAV vector expressing human AAT (rAAV1 …

Recombinant adeno-associated viral (rAAV) vectors based on serotype 2 are currently being
evaluated most extensively in animals and human clinical trials. rAAV vectors constructed …