• Designer Molecule Points to Treatment for Diseases Caused by DNA Repeats

    files/Filename/Aseem Ansari copy 2.jpg

    Using a molecule designed to overcome a roadblock formed by a common type of genetic flaw, researchers at the University of Wisconsin–Madison have made progress towards novel molecular treatments for Friedreich’s ataxia — a rare but fatal disorder — in the laboratory dish and in animals.

    Friedreich’s, like at least 40 other genetic diseases, is caused by stretches of repetitive DNA that prevent protein from forming correctly.

    The repeats can contain hundreds of identical, short sequences of DNA (such as GAAGAAGAAGAA …). In some diseases, including Friedreich’s, the repeats become roadblocks to cellular machines that decode the gene and start making the protein that the cell needs. In other diseases, such as the neurological condition Huntington’s, the repeats can result in excess protein, which itself can become toxic.

    In research reported this week in the journal ScienceAseem Ansari, a professor of biochemistry and genomics at UW–Madison, and colleagues showed that their “molecular prosthesis” can help cellular machinery overcome the blockade posed by the repeats in Friedreich’s ataxia.

    Read more about this research in the press release at the link below. 

    URL: https://biochem.wisc.edu/news/2017/news-ansari-ataxia-2017-11-30

    Date Published: Nov 30, 2017