Article
Reversal of aberrant splicing of beta-thalassemia allele by antisense RNA in vitro and in vivo.
Chinese medical journal - 1 Feb 1999
Zeng Y, Gu X, Chen Y, Gong L, Ren Z, Huang S
Abstract excerpt
OBJECTIVE: To investigate the reversal of aberrant splicing of beta-thalassemia allele (IVS-2-654 C-->T, beta 654) by antisense RNA in vitro and in vivo. METHODS: The vector expressing antisense RNA which targeted against the aberrant splice sites of beta 654 pre-mRNA was constructed in pcDNA3, and then used to repair the defective splicing of the mutant pre-mRNA in an in vitro transcription and splicing system,...
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