Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

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

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 …

Recombinant adeno-associated viral (AAV) vectors have rapidly advanced to the forefront of
gene therapy in the past decade. The exponential progress of AAV-based vectors has been …

Adeno-associated virus (AAV) is the leading vector in emerging treatments of inherited
diseases. Higher transduction efficiencies and cellular specificity are required for broader …

The structure of the adeno-associated virus (AAV-2) has been determined to 3-Å resolution
by x-ray crystallography. AAV is being developed as a vector for gene therapy to treat …

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 …

The serotypes of adeno-associated virus (AAV) have the potential to become important
resources for clinical gene therapy. In an effort to compare the role of serotype-specific virion …

▪ Abstract Broadly defined, the concept of gene therapy involves the transfer of genetic
material into a cell, tissue, or whole organ, with the goal of curing a disease or at least …

Infection of cells with adeno-associated virus (AAV) type 2 (AAV-2) is mediated by binding to
heparan sulfate proteoglycan and can be competed by heparin. Mutational analysis of AAV …