Many neurological diseases are caused by pathogenic unstable intragenic trinucleotide repeat (TNR) expansions, such as the GAA trinucleotide repeat within the FXN gene in Friedreich ataxia (FRDA) and ...
Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington’s disease and Friedreich’s ataxia. The conditions are two of more than 40 severe neurological ...
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