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METHOD FOR RETAINING SPLICING RNAS IN THE NUCLEUS BASED ON CHEMICALLY MODIFIED ANTISENSE OLIGONUCLEOTIDES
METHOD FOR RETAINING SPLICING RNAS IN THE NUCLEUS BASED ON CHEMICALLY MODIFIED ANTISENSE OLIGONUCLEOTIDES
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机译:基于化学改性的反义寡核苷酸在核中保持RNA的剪切RNA
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摘要
The inventive technology relates to systems and methods for the use of chemically modified antisense oligonucleotides (ASOs) to sterically prevent intron excision thus causing nuclear retention of the RNA and inhibiting nuclear export. In preferred therapeutic embodiments, ASOs may be engineered to quarantine clinically relevant RNAs in the nucleus of the cell.
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