Hunting out the repeat expansion in Huntington's pigs

A recent study from Yan et al.(2023)provides the milestone evi-dence in the large animal model demonstrating genome edit-ing as a potential therapeutic strategy for Huntington's disease treatment. Huntington's disease(HD)is a devastating monogenetic neuro-degenera tive disorder that is caused by an expanded CAG repeat in the exon 1 of the HTT gene located in the 4th chromosome of the human genome(Macdonald,1993).The prevalence of HD is～1 out of 10,000 and carrier of the pathogenic mutant gene is many times more than this(Gardiner et al.,2019).