- 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...
Expand abstract - Publisher copy:
- 10.1038/ng0498-378
-
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- 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
- Language:
- English
- Keywords:
- Copyright date:
- 1998
Journal article
Ribozyme-mediated trans-splicing of a trinucleotide repeat.
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