Article
Engineered mischarged transfer RNAs for correcting pathogenic missense mutations.
Molecular therapy : the journal of the American Society of Gene Therapy - 7 Feb 2024
Hou Yichen, Zhang Wen, McGilvray Philip T, Sobczyk Marek, Wang Tianxin, Weng Shao Huan Samuel, Huff Allen, Huang Sihao, Pena Noah, Katanski Christopher D, Pan Tao
Abstract excerpt
Missense mutations account for approximately 50% of pathogenic mutations in human genetic diseases, and most lack effective treatments. Gene therapies, gene editing, and RNA therapies, including transfer RNA (tRNA) modalities, are common strategies for genetic disease treatments. However, reported tRNA therapies are for nonsense mutations only. It has not been explored how tRNAs can be engineered to correct...
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