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...
Κύριοι συγγραφείς: | Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC |
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Μορφή: | Journal article |
Γλώσσα: | English |
Έκδοση: |
Cell Press
2022
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Παρόμοια τεκμήρια
Παρόμοια τεκμήρια
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Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
ανά: Britt Hanson, κ.ά.
Έκδοση: (2022-12-01) -
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties
ανά: Chwalenia, K, κ.ά.
Έκδοση: (2022) -
Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice.
ανά: Dejia Li, κ.ά.
Έκδοση: (2010-12-01) -
Immunogold confirmation that utrophin is localized to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle.
ανά: Culle, M, κ.ά.
Έκδοση: (2001) -
Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
ανά: Goyenvalle, A, κ.ά.
Έκδοση: (2010)