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2. 发明申请

WO2009044294A2 MEANS AND METHODS FOR THE TREATMENT OF CATARACT AND PRESBYOPIA 审中-公开
标题翻译：用于治疗感染和预后的手段和方法
公开(公告)号：WO2009044294A2
公开(公告)日：2009-04-09
申请号：PCT/IB2008003602
申请日：2008-06-12
申请人： UNIV GENEVE , HOPITAUX UNIVERSITAIRES DE GEN , KRAUSE KARL-HEINZ , SERRANDER LENA
发明人： KRAUSE KARL-HEINZ , SERRANDER LENA
IPC分类号： C12N9/02 , A61K31/713 , A61P27/10 , A61P27/12 , C12N15/11 , C12Q1/26
CPC分类号： C12N9/0036 , A61K38/005
摘要： The present invention relates to an inhibitor of a protein, wherein said protein (i) comprises or consists of the amino acid sequence of SEQ ID NOs: 2 or 4, or is encoded by a nucleic acid comprising or consisting of the sequence of SEQ ID NOs: 1 or 3; (ii) comprises or consists of the amino acid sequence of SEQ ID NO: 26, or is encoded by a nucleic acid comprising or consisting of the sequence of SEQ ID NO: 25; (iii) is a fragment of the protein according to (i), or has a sequence at least 75% identical with the protein according to (i) or with said fragment of the protein according to (i), and exhibits NADPH oxidase activity; or (iv) is a fragment of the protein according to (ii) or has a sequence at least 75% identical with the protein according to (ii) or with said fragment of the protein according to (ii) and stabilizes said protein exhibiting NADPH oxidase activity; and/or an inhibitor of a nucleic acid, wherein said nucleic (v) comprises or consists of the nucleic acid sequence of SEQ ID NO: 1 or 3, or encodes a protein comprising or consisting of the sequence of SEQ ID NO: 2 or 4; (vi) comprises or consists of the nucleic acid sequence of SEQ ID NO: 25, or encodes a protein comprising or consisting of the sequence of SEQ ID NO: 26; (vii) is a fragment of the nucleic acid according to (v), or has a sequence at least 75% identical with the nucleic acid according to (v) or with said fragment of the nucleic acid according to (v), and encodes a protein exhibiting NADPH oxidase activity; or (viii) is a fragment of the nucleic acid according to (vi), or has a sequence at least 75% identical with the nucleic acid according to (vi) or with said fragment of the nucleic acid according to (vi), and encodes a protein stabilizing said protein exhibiting NADPH oxidase activity; for treatment and/or prevention of cataract and/or presbyopia. The sequences define NADPH oxidases or a subunit of an NADPH oxidase, respectively. A preferred inhibitor is a siRNA. Also provided are a method of treating cataract and presbyopia, pharmaceutical compositions, medical uses and diagnostic uses of compounds of the invention.
摘要翻译：本发明涉及蛋白质的抑制剂，其中所述蛋白质（i）包含SEQ ID NO：2或4的氨基酸序列或由SEQ ID NO：2或4的氨基酸序列组成，或由包含SEQ ID NO： NO：1或3; （ii）包含SEQ ID NO：26的氨基酸序列或由SEQ ID NO：26的氨基酸序列组成，或由包含或由SEQ ID NO：25的序列组成的核酸编码; （iii）是根据（i）的蛋白质的片段，或具有与根据（i）的蛋白质或根据（i）的蛋白质的所述片段至少75％相同的序列，并且表现出NADPH氧化酶活性 ; 或（iv）是根据（ii）的蛋白质的片段或具有与根据（ii）的蛋白质或根据（ii）的蛋白质的所述片段至少75％相同的序列，并稳定所述表达NADPH的蛋白质氧化酶活性; 和/或核酸的抑制剂，其中所述核酸（v）包含SEQ ID NO：1或3的核酸序列或由SEQ ID NO：1或3的核酸序列组成，或编码包含SEQ ID NO：2或SEQ ID NO：2的序列或由SEQ ID NO： 4; （vi）包含SEQ ID NO：25的核酸序列或由SEQ ID NO：25的核酸序列组成，或编码包含SEQ ID NO：26的序列或由SEQ ID NO：26的序列组成的蛋白质。（vii）是根据（v）的核酸的片段，或具有与根据（v）的核酸或根据（v）的核酸的所述片段至少75％相同的序列，并编码表现出NADPH氧化酶活性的蛋白质; 或（viii）是根据（vi）的核酸的片段，或具有与根据（vi）的核酸或根据（vi）的核酸的所述片段至少75％相同的序列，和编码稳定表达NADPH氧化酶活性的所述蛋白质的蛋白质; 用于治疗和/或预防白内障和/或老花眼。序列分别定义NADPH氧化酶或NADPH氧化酶的亚基。优选的抑制剂是siRNA。还提供了治疗本发明化合物的白内障和老花眼的方法，药物组合物，医学用途和诊断用途。

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