For central nervous system (CNS) applications, attempts are being made to deliver AAV gene therapy directly to the brain parenchyma or the cerebrospinal fluid (CSF) space by intra-cisterna magna ...

Somatic gene transfer with vectors such as adeno-associated virus (AAV) can overcome these limitations. ReGenX's AAV Vector Technology gives extremely efficient and stable gene expression in ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Significant variability exists in adeno-associated virus (AAV) measurement methods, especially with polymerase chain reaction-enzyme linked immunosorbent assay (PCR-ELISA), highlighting the need ...

NanoCas, an ultracompact nuclease, enables efficient muscle editing in NHPs using a single AAV - advancing gene editing for hard-to-target tissues.