The mouse model closely mimics the specific dysferlin mutation and resulting disease, and enabled the researchers to evaluate how the complete therapy works—taking muscle stem cells, correcting ...
2005). Age-dependent increase in total tau measured in the cerebral spinal fluid (personal communication, reMYND). These transgenic mice overexpress human tau, specifically the 4R/2N isoform, bearing ...
"The mutation-containing HSCs give rise to proinflammatory ... to the bad proteins that hurt normal cells." The LJI team used mouse models with mutated ASXL1 to solve this mystery.