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Tissue-specific peptide conjugates and methods
专利权人:
サレプタ セラピューティクス インコーポレイテッド
发明人:
モールトン ホン エム.,アイバーセン パトリック エル.
申请号:
JP2015182739
公开号:
JP6584888B2
申请日:
2015.09.16
申请国别(地区):
JP
年份:
2019
代理人:
摘要:
Cell-penetrating peptides useful for targeting a therapeutic compound to a selected mammalian tissue, methods for their identification, methods of forming conjugate compounds containing such peptides, and conjugates formed thereby are disclosed. The cell-penetrating peptides are 8 to 30 amino acid residues in length and consist of subsequences selected from the group consisting of RXR, RX, RB, and RBR; where R is arginine, B is ²-alanine, and each X is independently -C(O)-(CHR 1 ) n -NH-, where n is 4-6 and each R 1 is independently H or methyl, such that at most two R 1 's are methyl. In one embodiment, X is a 6-aminohexanoic acid residue.
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