Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

Adeno-associated virus (AAV) has emerged as a leading platform for gene delivery for
treating various diseases due to its excellent safety profile and efficient transduction to …

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 …

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

Background In patients with severe hemophilia B, gene therapy that is mediated by a novel
self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to …

In the last decade, enormous progress has been made in the development of gene therapy
for hemophilia A and B. After the first encouraging results of intravenously administered …

Glaucoma is a leading cause of irreversible blindness worldwide and is characterized by a
slow, progressive, and multifactorial degeneration of retinal ganglion cells (RGCs) and their …

Highlights•AAV is a non-pathogenic virus, and recombinant AAV vectors have proven to be
highly efficient for gene delivery to a wide variety of cell types, tissue, and organs in small …

An obstacle to cancer immunotherapy has been that the affinity of T-cell receptors (TCRs) for
antigens expressed in tumors is generally low. We initiated clinical testing of engineered T …

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 …