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Platform of engineered tRNAs that target and suppress disease caused by nonsense mutations

Background Nonsense mutations, or premature termination codons (PTCs), make up 10-15% of all genetic diseases, affecting over 30 million people worldwide through 7,000 different diseases. Currently, there are very limited options for their therapeutic management. Some of the available agents have significant issues such as off-target toxicity and low...

Published: 8/14/2023 | Inventor(s): John Lueck, Joseph Porter, Charles Thornton, Wooree Ko

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Category(s): Research Tools