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 …

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 …

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 …

Host immune responses that limit durable therapeutic gene expression and cause clinically
significant inflammation remain a major barrier to broadly successful development of adeno …

Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …

Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible cure for
hemophilia. However, immune responses directed against AAV vectors remain a hurdle to …

Recombinant adeno-associated virus (rAAV) has attracted a significant research focus for
delivering genetic therapies to target cells. This non-enveloped virus has been trialed in …

Peripheral artery disease (PAD) is a vascular disorder caused by occlusive atherosclerosis,
which commonly impairs blood flow to the lower extremities. The prevalence of PAD is …

Pompe disease (PD) is a neuromuscular disorder caused by acid α‐glucosidase (GAA)
deficiency. Reduced GAA activity leads to pathological glycogen accumulation in cardiac …

Gene therapy using adeno-associated viral (AAV) vectors is a promising therapeutic strategy
for multiple inherited diseases. Following intravenous injection, AAV vectors carrying a copy …