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) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …

Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

Several viral vector-based gene therapy drugs have now received marketing approval. A
much larger number of additional viral vectors are in various stages of clinical trials for the …

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

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 …

Macrophages are essential for skeletal muscle homeostasis, but how their dysregulation
contributes to the development of fibrosis in muscle disease remains unclear. Here, we used …

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

Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo
gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer …

In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …