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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

Authors


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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
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

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