Clinical gene therapy has been increasingly successful owing both to an enhanced
molecular understanding of human disease and to progressively improving gene delivery …

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 …

Efficient retrograde access to projection neurons for the delivery of sensors and effectors
constitutes an important and enabling capability for neural circuit dissection. Such an …

In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles
for clinical gene therapy, particularly for monogenic recessive disorders, but there has also …

Systemic injection into blood vessels is the most common method of drug administration.
However, targeting drugs to the heart is challenging, owing to its dynamic mechanical …

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

Existing recombinant adeno-associated virus (rAAV) serotypes for delivering in vivo gene
therapy treatments for human liver diseases have not yielded combined high-level human …

Gene delivery vectors based on adeno-associated virus (AAV) have been utilized in a large
number of gene therapy clinical trials, which have demonstrated their strong safety profile …

Gene therapy is a promising approach for treating a spectrum of neurological and
neurodegenerative disorders by delivering corrective genes to the central nervous system …

More than one hundred naturally occurring variants of adeno-associated virus (AAV) have
been identified, and this library has been further expanded by an array of techniques for …