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 …

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

Work in animals and humans has suggested the existence of a slow wave sleep (SWS)-
promoting/electroencephalogram (EEG)-synchronizing center in the mammalian lower …

Recombinant adeno-associated virus (rAAV) vectors offer promise for the gene therapy of α1-
antitrypsin (AAT) deficiency. In our prior trial, an rAAV vector expressing human AAT (rAAV1 …

It has been recently shown that recombinant adeno-associated virus serotype 8 (rAAV8) is a
robust alternative serotype vector that overcomes many of the limitations of rAAV2 and …

Adeno-associated virus (AAV) based vectors have emerged as important tools for gene
therapy in humans. The recent successes seen in Phase I/II clinical trials have also …

Recombinant adeno-associated virus (AAV) is a promising delivery vehicle for in vivo gene
therapy and has been widely used in> 200 clinical trials globally. There are already several …

Alzheimer's disease (AD) is a progressive degenerative neurological disorder affecting
nearly one in nine elderly people in the United States. Population studies have shown that …

Late infantile Batten disease (CLN2 disease) is an autosomal recessive, neurodegenerative
lysosomal storage disease caused by mutations in the CLN2 gene encoding tripeptidyl …

The treatment of the more than 1,800 known monogenic hereditary disorders will depend on
the development of'genetic medicines'—therapies that use the transfer of DNA and/or RNA …