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IMPROVEMENTS IN ANTISENSE OLIGOMERS, DELIVERY OF ANTISENSE OLIGOMERS, AND IDENTIFICATION OF ANTISENSE OLIGOMER TARGETS
IMPROVEMENTS IN ANTISENSE OLIGOMERS, DELIVERY OF ANTISENSE OLIGOMERS, AND IDENTIFICATION OF ANTISENSE OLIGOMER TARGETS
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机译:反义寡聚物的改进,反义寡聚物的交付以及反义寡聚物目标的确定
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摘要
Antisense oligomers which possess improved properties over those taught in the prior art are disclosed. Preferably, oligomers for delivery to a cell comprising at least one nucleomonomer having a silicon-based 2'OH protecting group, e.g., a tertbutyl dimethyl-silyl group. Oligomers of between about 7 and about 12 nucleomonomers in length which are complementary to a site on an RNA target molecule selected from the group consisting of: the extreme 5' terminus of the RNA molecule, a 5' splice junction of the RNA molecule, and a 3' splice junction of the RNA molecule are provided. Oligomers comprising a neutral backbone which have been modified to comprise at least one charged group are also disclosed. In addition, methods of identifying an oligomer complementary to a cellular gene involved in a physiological process are provided.
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