Tardbp^em1H

Using CRISPR/Cas9 technology with sgRNA and ssODN donor template, a c.991C>A mutation was created that results in the amino acid substitution of glutamine with lysine at position 331 (p.Q331K). This mutation mimics the same mutation in human amyotrophic lateral sclerosis (ALS) patients. The mutation is located in the low complexity domain (LCD) of the encoded peptide. (J:265666)