Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9
(2018)
Journal Article
Koo, T., Lu-Nguyen, N. B., Malerba, A., Kim, E., Kim, D., Cappellari, O., Cho, H.-Y., Dickson, G., Popplewell, L., & Kim, J.-S. (2018). Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9. Molecular Therapy, https://doi.org/10.1016/j.ymthe.2018.03.018
Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle wasting disease caused by mutations in the DMD gene. In 51% of DMD cases, a reading frame is disrupted because of deletion of several exons. Here, we show that CjCas9 derived from Campylob... Read More about Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9.