Recombinant Adeno-associated virus (rAAV) is one of the main delivery vectors for gene
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

Background The prevention of bleeding with adequately sustained levels of clotting factor,
after a single therapeutic intervention and without the need for further medical intervention …

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 …

Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

Gene therapy products for the treatment of genetic diseases are currently in clinical trials,
and one of these, an adeno-associated viral (AAV) product, has recently been licensed. AAV …

In vivo gene replacement for the treatment of inherited disease is one of the most compelling
concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively …

The unique life cycle of adeno-associated virus (AAV) and its ability to infect both
nondividing and dividing cells with persistent expression have made it an attractive vector …

Conventional methods for rAAV purification that are based on cesium chloride
ultracentrifugation have often produced vector preparations of variable quality and resulted …

The efficient delivery of therapeutic genes and appropriate gene expression are the crucial
issues for clinically relevant gene therapy. Viruses are naturally evolved vehicles which …