Unnatural Amino Acid‐Based Ionic Liquid Enables Oral Treatment of Nonsense Mutation Disease in Mice
Yujie Shi,
Ningning Shi,
Yuelin Yang
et al.
Abstract:This investigation addresses the challenge of suboptimal unnatural amino acid (UAA) utilization in the site‐specific suppression of nonsense mutations through genetic code expansion, which is crucial for protein restoration and precise property tailoring. A facile and economical oral liquid formulation is developed by converting UAAs into ionic liquids, significantly enhancing their bioavailability and tissue accumulation. Empirical data reveal a 10‐fold increase in bioavailability and up to a 13‐fold rise in … Show more
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