Mutant allele-specific CRISPR disruption in DYT1 dystonia fibroblasts restores cell function

by Cruz, Lilian, Bence, György, Cheah, Pike See, Kleinstiver, Benjamin P., Eimer, William A., Garcia, Sara P., Sharma, Nutan, Ozelius, Laurie J., Cristopher, Bragg D., Joung, Keith J., Norberto de Souza, Osmar, Macedo Timmers, Luis Fernando Saraiva, Breakefield, Xandra O.
Published 2020