Gene therapy using adeno-associated viruses (AAVs) is transforming medicine by treating previously incurable genetic disorders. However, verifying the quality of these therapies remains challenging. Current methods for checking AAV DNA are inadequate – they're either too imprecise to detect important genetic changes or too slow and expensive for routine use.
This project develops quality control techniques using long-read DNA sequencing technology from Oxford Nanopore Technologies and Pacific Biosciences. Unlike current methods, these platforms can read entire AAV genomes in a single pass, revealing crucial details about their structure and composition.
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Johns Hopkins University
