Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …

Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. The
benefits of using AAV for gene therapy include long-term gene expression, the inability to …

Recombinant adeno-associated viral vectors (rAAVs) have been widely used for gene
delivery in animal models, and are currently evaluated for human gene therapy after …

Abstract The Adeno-associated viruses (AAVs) are not associated with any diseases, and
their ability to package non-genomic DNA and to transduce different cell/tissue populations …

Decades ago, Friedmann and Roblin postulated several barriers to gene therapy, including
tissue targeting, delivery across the blood–brain barrier (BBB), and host immune responses …

We have documented that epidermal growth factor receptor protein tyrosine kinase (EGFR-
PTK) signaling negatively affects intracellular trafficking and transduction efficiency of …

Recent clinical trials have shown that evasion of CD8+ T-cell responses against viral capsid
is critical for successful liver-directed gene therapy with adeno-associated viral (AAV) …

Adeno-associated virus (AAV) vectors are effective gene delivery vehicles mediating long-
lasting transgene expression. Data from a clinical trial of AAV2-mediated hepatic transfer of …

Adeno-associated virus (AAV) is a replication-deficient parvovirus that is extensively used as
a gene therapy vector. CD8+ T-cell responses against the AAV capsid protein can, however …

Highlights•Engineering of AAV capsids optimizes efficiency and/or specificity of gene
transfer.•High throughput-selection of AAV libraries identifies AAV variants with novel …