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...
Үндсэн зохиолчид: | Hanson, B, Stenler, S, Ahlskog, N, Chwalenia, K, Svrzikapa, N, Coenen-Stass, AML, Weinberg, MS, Wood, MJA, Roberts, TC |
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Формат: | Journal article |
Хэл сонгох: | English |
Хэвлэсэн: |
Cell Press
2022
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Ижил төстэй зүйлс
Ижил төстэй зүйлс
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Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD
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Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice.
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Immunogold confirmation that utrophin is localized to the normal position of dystrophin in dystrophin-negative transgenic mouse muscle.
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Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
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