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 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 …

Autosomal recessive genetic forms (DFNB) account for most cases of profound congenital
deafness. Adeno-associated virus (AAV)-based gene therapy is a promising therapeutic …

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 …

The ability to elicit robust and long-term transgene expression in vivo together with minimal
immunogenicity and little to no toxicity are only a few features that make recombinant adeno …

Adeno-associated virus (AAV) has shown great promise as a gene therapy vector in multiple
aspects of preclinical and clinical applications. Many developments including new serotypes …

The HEK293 cell line has earned its place as a producer of biotherapeutics. In addition to its
ease of growth in serum-free suspension culture and its amenability to transfection, this cell …

Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal
diseases that are incurable. One molecular form is caused by mutations in the RPE65 …

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 …

Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes to
neurons effectively in the brain, retina, and spinal cord. The characterization of new AAV …