In this report we propose to define the benchmark for the ideal rAAV as the specific infectivity of wild-type AAV (wtAAV). The vp-to-iu ratio of the rAAV2-GFPa was determined to be 124 (Table 1 ...

EGFP was expressed in neurons in the hippocampi of 7-month-old mice injected with AAV-EGFP-sTREM2 or AAV-EGFP four months previously ... compared with 5xFAD mice expressing wild-type Trem2. This ...

This study provides important insights in the structure/function of human wild-type AAV capsids and support the therapeutic potential of natural AAV capsids for central nervous system and muscle ...

Another major goal of gene therapy research is improving AAV tissue specificity and augmenting tissue transduction, i.e., that the AAVs target the intended tissue and disseminate widely across it.

Adeno-associated virus (AAV)–mediated gene therapy has emerged ... in higher factor IX activity levels than a vector with the wild-type factor IX variant but in similar levels of factor IX ...

Advanced Medicine Partners’ process has been used with multiple genes of interest as well as multiple wild-type AAV serotypes and engineered capsids. This manufacturing process prioritizes ...