A team of Shihua LI at Emory University, Liangxue LAI at CAS South China Institute of Stem Cells and Regeneration Medicine, and Sen YAN at Jinan University used the CRISPR/Cas9 gene editing technique to introduce a segment of a human gene causing Huntington’s, with a very long glutamine repeat region, into pig fibroblast cells, Somatic cell nuclear transfer generated pig embryos carrying this genetic alteration. The “knock in” technique changing a gene in its natural sequence led to pigs which displayed symptoms such as movement problems, respiratory difficulties, which resemble those experienced by humans with HD and degeneration of the striatum, the region of the brain most affected by HD in humans, more than other regions of the brain. The pig model more closely matches the symptoms of the human disease, and treatments to affected nervous system tissues can be better tested in pigs compared to mice.

CAS news release, March 30, 2018