Báo cáo khoa học: Trans-splicing of a mutated glycosylasparaginase mRNA sequence by a group I ribozyme deficient in hydrolysis
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RNAreprogramming represents anewconcept incorrecting genetic defects at theRNA level.However, for the technique to be useful for therapy, the level of reprogrammingmust be appropriate. To improve the efficiency of group I ribozyme-mediated RNA reprogramming, when using theTetrahy-menaribozyme, regions complementary to the target RNA have previously been extended in length and accessible sites in the targetRNAs have been identified.
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