In recent years, the number of clinical trials in which adeno-associated virus (AAV) vectors
have been used for in vivo gene transfer has steadily increased. The excellent safety profile …

Recombinant adeno-associated virus (rAAV) vectors are one of the most promising in vivo
gene delivery tools. Several features make rAAV vectors an ideal platform for gene transfer …

Recombinant adeno-associated viruses (rAAVs) are commonly used vehicles for in vivo
gene transfer 1, 2, 3, 4, 5, 6. However, the tropism repertoire of naturally occurring AAVs is …

We recently developed adeno-associated virus (AAV) capsids to facilitate efficient and
noninvasive gene transfer to the central and peripheral nervous systems. However, a …

Gene therapy products for the treatment of genetic diseases are currently in clinical trials,
and one of these, an adeno-associated viral (AAV) product, has recently been licensed. AAV …

Prevalence of type 2 diabetes (T2D) and obesity is increasing worldwide. Currently
available therapies are not suited for all patients in the heterogeneous obese/T2D …

Gene therapy mediated by recombinant adeno-associated virus (AAV) vectors is a
promising treatment for systemic monogenic diseases. However, vector immunogenicity …

Site-specific genome editing provides a promising approach for achieving long-term, stable
therapeutic gene expression. Genome editing has been successfully applied in a variety of …

A major goal in aging research is to improve health during aging. In the case of mice,
genetic manipulations that shorten or lengthen telomeres result, respectively, in decreased …

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 …