David Liu, recognized for his contributions to gene-editing technology, plays a pivotal role in advancing treatments for genetic diseases. The breakthrough case of Alyssa Tapley, a 13-year-old leukemia patient, showcases the success of base editing that cleared her cancer. Liu emphasizes the responsibility that comes with developing these therapies, aiming for safety and effectiveness. His work transcends the traditional CRISPR-Cas9 methods, focusing instead on precise gene correction rather than just disruption. This evolution in gene technology opens new doors for millions suffering from genetic ailments, reflecting the essence of basic science.
It's incredibly exciting, and also comes with a heavy sense of responsibility, to make sure that - to the extent humanly possible - we have done everything we can to make these agents as safe and effective as possible for use in patients.
That approach of cutting the DNA double helix is very useful for gene disruption or deletion. But if your goal is to correct a mutation that causes a genetic disease, it's not easy to use scissors to achieve gene correction.
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