When CAG nucleotide triplets repeat excessively in a gene called huntingtin, a devastating and currently untreatable neurodegenerative “repeat expansion disorder” arises called Huntington’s Disease ...
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 ...
Armed with base editors, scientists from the laboratory of David Liu, PhD, professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute, and their ...
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