Journal article
Genetic therapies for RNA mis-splicing diseases.
- 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...
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- Publication status:
- Published
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- Publisher copy:
- 10.1016/j.tig.2011.02.004
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Authors
Bibliographic Details
- Journal:
- Trends in genetics : TIG
- Volume:
- 27
- Issue:
- 5
- Pages:
- 196-205
- Publication date:
- 2011-05-01
- DOI:
- ISSN:
-
0168-9525
- Source identifiers:
-
134947
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:134947
- UUID:
-
uuid:6a47d479-577c-4468-9f22-db629fc9b9ee
- Local pid:
- pubs:134947
- Deposit date:
- 2012-12-19
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- Copyright date:
- 2011
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