- Abstract:
-
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 disease-causing mutations. During the past 10years, genetic therapy directed toward correction of RNA mis-splicing in disease has progressed from theoretical work in cultured cells to promising clinic...
Expand abstract - Publication status:
- Published
- Publisher copy:
- 10.1016/j.tig.2011.02.004
-
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- Journal:
- Trends in genetics : TIG
- Volume:
- 27
- Issue:
- 5
- Pages:
- 196-205
- Publication date:
- 2011-05-05
- DOI:
- ISSN:
-
0168-9525
- URN:
-
uuid:6a47d479-577c-4468-9f22-db629fc9b9ee
- Source identifiers:
-
134947
- Local pid:
- pubs:134947
- Language:
- English
- Keywords:
- Copyright date:
- 2011
Journal article
Genetic therapies for RNA mis-splicing diseases.
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