Animal cell-based expression platforms enable the production of complex biomolecules
such as recombinant proteins and viral vectors. Although most biotherapeutics are produced …

Intracellular delivery is a key step in biological research and has enabled decades of
biomedical discoveries. It is also becoming increasingly important in industrial and medical …

Dominant missense pathogenic variants in cardiac myosin heavy chain cause hypertrophic
cardiomyopathy (HCM), a currently incurable disorder that increases risk for stroke, heart …

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 …

Neurotropic adeno-associated virus (AAV) serotypes such as AAV9 have been
demonstrated to transduce spinal alpha motor neurons when administered intravenously (iv) …

Exosomes can deliver therapeutic RNAs to neurons. The composition and the safety profile
of exosomes depends on the type of the exosome-producing cell. Mesenchymal stem cells …

Improved delivery of adeno-associated virus (AAV) vectors to the CNS will greatly enhance
their clinical utility. Selection of AAV9 variants in a mouse model led to the isolation of a …

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

Many genetic liver diseases in newborns cause repeated, often lethal, metabolic crises.
Gene therapy using nonintegrating viruses such as adeno-associated virus (AAV) is not …

Understanding the structure-function relationships at cellular, circuit, and organ-wide scale
requires 3D anatomical and phenotypical maps, currently unavailable for many organs …