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 …

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 …

Adeno-associated viruses (AAVs) are increasingly used as gene therapy vectors. AAVs
package their genome in a non-enveloped T= 1 icosahedral capsid of~ 3.8 megaDalton …

Charge detection mass spectrometry (CDMS) is a single-particle technique where the
masses of individual ions are determined from simultaneous measurement of their mass-to …

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

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 …

Efficient and widespread gene transfer is required for successful treatment of Duchenne
muscular dystrophy (DMD). Here, we performed the first clinical trial using a chimeric adeno …

Nonalcoholic steatohepatitis (NASH) is a progressive disease that is often accompanied by
metabolic syndrome and poses a high risk of severe liver damage. However, no effective …

Background Neurodevelopmental disorders, as a class of diseases, have been particularly
difficult to treat even when the underlying cause (s), such as genetic alterations, are …

Activation of apoptosis signal-regulating kinase 1 (ASK1) in hepatocytes is a key process in
the progression of nonalcoholic steatohepatitis (NASH) and a promising target for treatment …