Expanded repetitive sequences are infamous for causing inherited disease. Think poly-Q tracts that cause Huntington’s or the C9orf72 hexanucleotide run-ons that trigger ALS/FTD. However, given people ...
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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