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A method for treating a myotonic disorder

Background Myotonic disorders are a group of genetic diseases characterized by the presence of myotonia, which is defined as failure of muscle relaxation after activation. The presentation of these disorders can range from asymptomatic electrical myotonia, as seen in some forms of myotonia congenita (MC), to severe disability with muscle weakness,...

Published: 8/14/2023 | Inventor(s): John Lueck, Charles Thornton, Lily Cisco

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Category(s): Therapeutic

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