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

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 …

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 …

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) is the leading vector in emerging treatments of inherited
diseases. Higher transduction efficiencies and cellular specificity are required for broader …

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) serotypes differ broadly in transduction efficacies and tissue
tropisms and thus hold enormous potential as vectors for human gene therapy. In reality …

Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of
devastating lysosomal storage disorders that collectively represent the most common …

Adeno-associated virus (AAV) forms the basis for several commercial gene therapy products
and for countless gene transfer vectors derived from natural or synthetic viral isolates that …

Adeno-associated viral vectors are highly safe and efficient gene delivery vehicles.
However, numerous challenges in vector design remain, including neutralizing antibody …