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 …

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-
associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential …

Recombinant AAV vectors have been, or are currently in use, in 331 Phase I/II/III clinical
trials in a number of human diseases, and in some cases, remarkable clinical efficacy has …

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 …

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 …

Parvoviruses, infecting vertebrates and invertebrates, are a family of single-stranded DNA
viruses with small, non-enveloped capsids with T= 1 icosahedral symmetry. A quarter of a …

Adeno-associated viral (AAV) vectors have emerged as the leading gene delivery platform
for gene therapy and vaccination. Three AAV-based gene therapy drugs, Glybera …

Recombinant adeno-associated virus 2 (AAV2) vectors are in use in several Phase I/II
clinical trials, but relatively large vector doses are needed to achieve therapeutic benefits …

Vectors derived from adeno-associated viruses (AAVs) have become important gene
delivery tools for the treatment of many inherited ocular diseases in well-characterized …

Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …