Next-generation RNA approach provides hope for genetic diseases missed by current drugs

Researchers at the University of Toronto have developed a next-generation RNA therapeutic approach with the potential to treat a wide range of genetic diseases that share certain disease-causing mutations. The work advances an emerging platform in genetic medicine centered on transfer RNA, or tRNA. The team engineered tRNA to help cells read through premature stop signals and complete production of full-length proteins that would otherwise be truncated or absent.

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