초록 |
Adeno-associated viral (AAV) vectors have recently emerged as efficient therapeutic tools for treating rare diseases. As AAV-related technology and AAV-based therapeutics have been developed in both academia and industry, and, more importantly, AAV-based therapeutics have been approved by healthcare administrations in many countries, the efficient production of the vectors became of critical importance. However, among numerous steps and components in the AAV production process, purification has been a bottleneck since the conventional method has several limitations, requiring the necessity of special equipment such as ultracentrifugation or specialized filtration apparatus. Herein, a facile and serotype-independent microparticle-based AAV purification system is introduced. By modifying a common material utilized in protein purification columns into clickable counterparts of this protein, microparticles with high selectivity and strong affinity to multiple AAV serotypes have been developed. In this presentation, characterizations of the primary system and the potential of the presented system as an efficient AAV purification strategy are discussed. |