Conventional CRISPR nucleases, including Cas9 and Cas12a, exceed the packaging limits of a single AAV vector, necessitating ...

Gene therapy represents one of the fastest growing biotherapeutics worldwide, with AAV at the forefront. This rapid growth is constantly challenging CMC approaches, from manufacturing through release ...

In recent years, there has been an increase in the number of registered clinical trials evaluating recombinant adeno-associated virus (AAV)-based gene therapies, in part due to AAV’s ability to ...

Adeno-associated virus (AAV) vectors play a pivotal role in gene therapy, delivering therapeutic genes with high precision and safety. However, a significant challenge in the biomanufacturing of ...

To assist researchers in the selection of the optimal AAV vector for their application, we have generated a detailed map of viral delivery to tissues in mice, the most commonly used experimental ...