Genetic therapies for RNA mis-splicing diseases.
RNA mis-splicing diseases account for up to 15% of all inherited diseases, ranging from neurological to myogenic and metabolic disorders. With greatly increased genomic sequencing being performed for individual patients, the number of known mutations affecting splicing has risen to 50-60% of all dis...
| Main Authors: | , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2011
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