Novel gene therapy approach creates new route to tackle rare, inherited diseases | 2/19/2019 | Staff
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Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally, and nonsense mutations cause 10-15 percent of all inherited genetic diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis and polycystic kidney disease. There is currently no cure or broadly effective treatment for these often devastating conditions that are individually rare but estimated to collectively affect up to 30 million people worldwide.

A new study, led by Christopher Ahern, Ph.D., at the University of Iowa Carver College of Medicine, reveals a novel approach and robust technology platform for suppressing nonsense mutations using engineered transfer RNA (tRNA) molecules. The research by Ahern, his UI colleagues, and collaborators at The Wistar Institute in Philadelphia, the Cystic Fibrosis Foundation Therapeutics Lab in Lexington, Mass., and Integrated DNA Technologies Inc. in Coralville, Iowa, shows that modified tRNAs can efficiently and accurately repair nonsense mutations with any amino acid. The findings were published Feb. 18 in Nature Communications.

Mutations - Range - Life - Threatening - Diseases

"Because nonsense mutations cause a wide range of severe, life threatening diseases, there is a significant unmet medical need to efficiently repair these stop codons in people having these inherited genetic alterations," says Ahern, UI professor of molecular physiology and biophysics and a member of the Iowa Neuroscience Institute. "Our unique gene therapy approach takes advantage of the built-in fidelity of the translation process but reengineers tRNAs to turn disease-causing stop signals back into the correct amino acid. Basically, our anticodon engineered tRNA technology turns 'stops' into 'gos' and hopefully one day may be used to correct defective genetic sequences in people."

The process of turning genetic code into protein is called translation. Transfer RNAs (tRNAs) match up with the blueprint code of the messenger RNA and deliver the correct amino acid in the...
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