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METHOD FOR RETAINING SPLICING RNAS IN THE NUCLEUS BASED ON CHEMICALLY MODIFIED ANTISENSE OLIGONUCLEOTIDES

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

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.
机译:本发明的技术涉及用于使用化学改性的反义寡核苷酸(ASOS)的系统和方法,以防止内含子切除,从而导致核保留RNA并抑制核导出。在优选的治疗实施例中,可以在细胞的核中设计成在临床上检疫临床相关的RNA。

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