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Min, 2018 - Google Patents

Correction of Hot Spot Mutations in Duchenne Muscular Dystrophy by CRISPR/Cas9 Gene Editing

Min, 2018

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Document ID
17734303440803884975
Author
Min Y
Publication year

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More than 800 monogenic disorders result in degeneration or dysfunction of skeletal muscle (Bonne et al., 2017). Among the most severe is Duchenne muscular dystrophy (DMD), which is caused by mutations in the X-linked dystrophin gene, a massive gene spanning~ 2.3 …
Continue reading at utswmed-ir.tdl.org (PDF) (other versions)

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N5/06—Animal cells or tissues; Human cells or tissues ; Not used, see subgroups
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
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