How to turn muscle into a protein factory for advanced gene therapy

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How to turn muscle into a protein factory for advanced gene therapy

In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass Chan Medical School recently announced that they have mapped the expression and maturation of the protein alpha-1 antitrypsin (AAT) with unprecedented clarity. The results, which detail the molecular folding of the protein, were published in the Proceedings of the National Academy of Sciences and will help to develop specific therapies to treat an inherited disease known as alpha-1 antitrypsin deficiency, as well as more effectively treat a wide range of genetic diseases.

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