Wang, L. et. al. (2017) Protein Cell. https://www.ncbi.nlm.nih.gov/pubmed/28401346
ALS is a neurodegenerative disease, with mutations in SOD1 and FUS associated with familial ALS. Using CRISPR/Cas9 Wang et al. corrected human iPSCs generated from fibroblasts of familial ALS patients containing SOD1 and FUS mutations. These regenerated cells were used for transcriptome comparison studies to the mutated cells with close to 900 aberrant transcripts identified. Use of this system could lead to potential therapies for ALS patients with SOD1 and FUS mutations.