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 …

The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies
in Europe and the United States are landmark achievements in the history of modern …

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

Gene therapy for kidney diseases has proven challenging. Adeno-associated virus (AAV) is
used as a vector for gene therapy targeting other organs, with particular success …

Highlights•AAV is a non-pathogenic virus, and recombinant AAV vectors have proven to be
highly efficient for gene delivery to a wide variety of cell types, tissue, and organs in small …

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 adeno-associated viral (rAAV) vectors have shown early promise in clinical
trials,,. The therapeutic transgene cassette can be packaged in different AAV capsid …

The discovery of naturally occurring adeno-associated virus (AAV) isolates in different
animal species and the generation of engineered AAV strains using molecular genetics …

Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …

Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset
neuromuscular disorder characterized by progressive muscle weakness and atrophy …