Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …

The discovery of naturally occurring adeno-associated virus (AAV) isolates in different
animal species and the generation of engineered AAV strains using molecular genetics …

Recombinant gene delivery vectors derived from naturally occurring or genetically
engineered adeno-associated viruses (AAV) have taken center stage in human gene …

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

Viral vectors derived from adeno-associated viruses (AAVs) are widely used for gene
transfer both in vitro and in vivo. The increasing use of AAV as a gene transfer vector, as well …

Clinical gene therapy has been increasingly successful owing both to an enhanced
molecular understanding of human disease and to progressively improving gene delivery …

Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Adeno-Associated Virus based vectors (rAAV) are advantageous for human gene therapy
due to low inflammatory responses, lack of toxicity, natural persistence, and ability to …

Over the last two decades, gene therapy vectors based on wild‐type Adeno‐associated
viruses (AAV) are safe and efficacious in numerous clinical trials and are translated into …