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 …

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 …

There has been a resurgence in gene therapy efforts that is partly fueled by the identification
and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non …

Forming the inner layer of the vascular system, endothelial cells (ECs) facilitate a multitude
of crucial physiological processes throughout the body. Vascular ECs enable the vessel wall …

Adeno-associated virus (AAV) vectors are currently the leading candidates for virus-based
gene therapies because of their broad tissue tropism, non-pathogenic nature and low …

Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …

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 …

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

Both observational and perturbational technologies are essential for advancing the
understanding of brain function and dysfunction. But while observational techniques have …

Parvoviruses, infecting vertebrates and invertebrates, are a family of single-stranded DNA
viruses with small, non-enveloped capsids with T= 1 icosahedral symmetry. A quarter of a …