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 …

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 …

The human genetic code encrypted in thousands of genes holds the secret for synthesis of
proteins that drive all biological processes necessary for normal life and death. Though the …

Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …

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 …

Adeno-associated virus (AAV) vectors have emerged as a gene-delivery platform with
demonstrated safety and efficacy in a handful of clinical trials for monogenic disorders …

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 …

Background—Adeno-associated virus type 1/sarcoplasmic reticulum Ca2+-ATPase was
assessed in a randomized, double-blind, placebo-controlled, phase 2 study in patients with …

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 …

Despite tremendous efforts, development of an effective vaccine against human
immunodeficiency virus (HIV) has proved an elusive goal. Recently, however, numerous …