发明申请
- 专利标题: COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY
- 专利标题(中): 用于处理微电子显微镜的化合物和方法
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申请号: US13219401申请日: 2011-08-26
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公开(公告)号: US20120058946A1公开(公告)日: 2012-03-08
- 发明人: Hong M. Moulton , Ryszard Kole
- 申请人: Hong M. Moulton , Ryszard Kole
- 申请人地址: US OR Corvallis
- 专利权人: AVI BIOPHARMA, INC.
- 当前专利权人: AVI BIOPHARMA, INC.
- 当前专利权人地址: US OR Corvallis
- 主分类号: A61K38/08
- IPC分类号: A61K38/08 ; A61P9/00 ; A61P21/00 ; C07K7/02
摘要:
An antisense compound for use in treating myotonic dystrophy DM1 or DM2, a method of enhancing antisense targeting to heart and quadricep muscles, and a method for treating DM1 or DM2 in a mammalian subject are disclosed. The oligonucleotide has 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively. Conjugated to the oligonucleotide is a cell-penetrating peptide having the sequence (RXRR(B/X)R)2XB, where R is arginine; B is β-alanine; and each X is —C(O)—(CH2)n—NH—, where n is 4-6. The antisense compound is effective to selectively block the sequestration of muscleblind-like 1 protein (MBNL1) and/or CUGBP, in heart and quadricep muscle in a myotonic dystrophy animal model.
公开/授权文献
- US08741863B2 Compound and method for treating myotonic dystrophy 公开/授权日:2014-06-03
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