RNA Breakthrough Provides Hope for Thousands of Untreatable Diseases
7 Articles
7 Articles
A team of researchers has developed a new therapy based on state-of-the-art RNA that has the potential to treat a wide range of genetic diseases that share the mutations that cause them, from muscle dystrophy to some types of cancer.The finding, described this Thursday in Science magazine, is based on the so-called transfer RNA (RNAt), a small molecule of RNA that carries specific amino acids that play a fundamental role in protein synthesis.The…
One study shows the potential to attack a mechanism common to 11% of hereditary diseases
The so-called ‘senseless mutations’ (nonsense mutatis mutandis) are alterations in the genetic code that introduce a signal of premature stop during protein synthesis. As a result, cells produce inoperative versions of molecules fundamental to the organism. Although this type of genetic error represents approximately 11% of hereditary disorders, it is able to affect thousands of different pathologies, including variants of cystic fibrosis, muscu…
University of Toronto study advances tRNA approach for genetic diseases
Researchers at the University of Toronto have developed an RNA therapeutic approach that could treat genetic diseases linked to certain disease-causing mutations. The work advances an emerging platform in genetic medicine centred on transfer RNA, or tRNA. The team engineered tRNA to help cells read through premature stop signals and complete the production of full-length proteins that would otherwise be truncated or absent. Study lead Bowen Li, …
RNA breakthrough provides hope for thousands of untreatable diseases
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 centred 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 tru…
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