Advancing AAV genome integrity assessment with digital PCR

Gene therapy transforms how we approach genetic diseases, turning challenges in medicine into achievable goals. Among the most widely used tools in this field are adeno-associated virus (AAV) vectors, non-pathogenic carriers that enable stable delivery of therapeutic genes. Maintaining consistent viral vector quality across each production stage has become essential as more programs move toward clinical and commercial stages. One of the most critical aspects of AAV quality is assessing genome integrity, which directly influences potency and therapy efficacy.

When AAV genomes are incomplete or truncated, activity can be reduced. Conventional techniques such as gel electrophoresis, Southern blotting, or sequencing have provided valuable information, but they are often time-consuming and provide only limited quantitative precision for routine decision-making. Next-generation sequencing (NGS) has become a powerful tool for assessing AAV genome integrity, offering detailed insights into sequence composition and structural variations. While NGS excels in deep characterization, it is not optimized for rapid, high-throughput decisions, creating a need for complementary methods like digital PCR (dPCR). dPCR addresses these limitations by providing an accurate, sensitive, and reproducible way to determine whether AAV genomes are intact, fragmented, or likely to perform as intended.

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