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Ribozyme-mediated trans-splicing of a trinucleotide repeat.

Abstract:

Trinucleotide repeat expansions (TREs) are a recently described class of mutations characterized by a change in the size of the genomic fragment due to amplification of the repeated unit. A number of diseases have been attributed to TRE, including Huntington disease and myotonic dystrophy (DM), but attempts at genetic therapy have yet to prove successful. A potential therapeutic approach would be to repair the expanded repeat using the trans-splicing ability of group I intron ribozymes. We ha...

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Publisher copy:
10.1038/ng0498-378

Authors


Phylactou, LA More by this author
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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Journal:
Nature genetics
Volume:
18
Issue:
4
Pages:
378-381
Publication date:
1998-04-05
DOI:
EISSN:
1546-1718
ISSN:
1061-4036
URN:
uuid:2e1253d5-bc17-4619-8cc8-828402d6c352
Source identifiers:
114032
Local pid:
pubs:114032

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