SELECTIVE INHIBITION OF POLYGLUTAMINE PROTEIN EXPRESSION
摘要
The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntingtin Disease Protein and Ataxin-3 using double- stranded RNAs and nucleic acid analogs. Chemically-modified RNAs having at least one mismatch as compared to the target CAG repeat sequence are specifically contemplated.
申请公布号
WO2011097388(A1)
申请公布日期
2011.08.11
申请号
WO2011US23615
申请日期
2011.02.03
申请人
ALNYLAM PHARMACEUTICALS, INC.;THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM;COREY, DAVID, R.;HU, JIAXIN;SAH, DINAH, W., Y.
