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 ...

EULAR first wrote recommendations for small and medium vessel vasculitis in 2009; this was subsequently updated in 2016 with more of a focus on AAV. The new 2022 update includes substantial ...

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 ...

However, two large companion studies in non-human primates indicate that vector integrations in primate liver following AAV gene therapy may be an important mechanism for achieving durable ...

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 ...