Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy. [electronic resource]
Producer: 20140218Description: 702-13 p. digitalISSN:- 1557-7422
- Blotting, Western
- Calcium -- metabolism
- DNA Primers -- genetics
- Exons -- genetics
- Gene Expression Regulation -- genetics
- Genetic Therapy -- methods
- Genetic Vectors -- genetics
- HEK293 Cells
- Humans
- Lentivirus
- Microscopy, Fluorescence
- Mutation -- genetics
- Myopathy, Central Core -- therapy
- Reverse Transcriptase Polymerase Chain Reaction
- Ryanodine Receptor Calcium Release Channel -- genetics
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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