Sentences with phrase «patient myoblasts»

Addgene depositor Charles Gersbach used paired zinc finger nucleases to remove exon 51 in DMD patient myoblasts.

In a subsequent study, they used CRISPR with two gRNAs to delete exon 51 or exons 45 - 55 in patient myoblasts; when injected into DMD mice, these cells expressed functional dystrophin.

Not exact matches

Using myoblasts from DNM2 - mutated patients and using myoblasts and muscles from a knock - in mouse model of DNM2 - related myopathy, we analyzed structure of costameres by biochemical and immunocytochemical approaches, as well as their ultrastructure.

Modulation of Protein Quality Control and Proteasome to Autophagy Switch in Immortalized Myoblasts from Duchenne Muscular Dystrophy Patients.

He presented results from both SMA mouse models and patients that demonstrated SMN depletion results in muscle - intrinsic defects such as reduced satellite cell number, a delay in the myogenic program, reduced myoblast fusion, and molecular homeostasis disruption.