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 …

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 …

Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

Clinical gene therapy has been increasingly successful owing both to an enhanced
molecular understanding of human disease and to progressively improving gene delivery …

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 …

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 …

Therapeutic payload delivery to the central nervous system (CNS) remains a major
challenge in gene therapy. Recent studies using function-driven evolution of adeno …

Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years
ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species …

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 …

Fifty years after the discovery of adeno-associated virus (AAV) and more than 30 years after
the first gene transfer experiment was conducted, dozens of gene therapy clinical trials are …