Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
<div>Duchenne muscular dystrophy (DMD) is the most prevalent inherited myopathy affecting children, caused by genetic loss of the gene encoding the dystrophin protein. Here we have investigated the use of the <em>Staphylococcus aureus</em> CRISPR-Cas9 system and a...
Những tác giả chính: | Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC |
---|---|
Định dạng: | Journal article |
Ngôn ngữ: | English |
Được phát hành: |
Cell Press
2022
|
Những quyển sách tương tự
-
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
Bằng: Britt Hanson, et al.
Được phát hành: (2022-12-01) -
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
Bằng: Chwalenia, K, et al.
Được phát hành: (2022) -
Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice.
Bằng: Dejia Li, et al.
Được phát hành: (2010-12-01) -
Immunogold confirmation that utrophin is localized to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle.
Bằng: Culle, M, et al.
Được phát hành: (2001) -
Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
Bằng: Goyenvalle, A, et al.
Được phát hành: (2010)