Application of CRISPR-Cas9-mediated genome editing for the treatment of myotonic dystrophy type 1
Myotonic dystrophy type 1 (DM1) is a debilitating multisystemic disorder, caused by expansion of a CTG microsatellite repeat in the 3ʹ untranslated region of the DMPK (dystrophia myotonica protein kinase) gene. To date, novel therapeutic approaches have focused on transient suppression of the mutant...
| Main Authors: | , , , , |
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| Format: | Journal article |
| Language: | English |
| Published: |
Cell Press
2020
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