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1. 发明申请

WO2006081444A3 FARNESYLTRANSFERASE INHIBITORS FOR TREATMENT OF LAMINOPATHIES, CELLULAR AGING AND ATHEROSCLEROSIS 审中-公开
公开(公告)号：WO2006081444A3
公开(公告)日：2006-08-03
申请号：PCT/US2006/002977
申请日：2006-01-27
申请人： THE GOVERNMENT OF THE USA AS REPRESENTED BY THE SECRETARY OF THE DEPT. OF HEALTH AND HUMAN SERVICES , PROGERIA RESEARCH FOUNDATION , THE REGENTS OF THE UNIVERSITY OF MICHIGAN , THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL , GORDON, Leslie, B. , COLLINS, Francis, S. , GLOVER, Thomas , GLYNN, Michael, W. , CAPELL, Brian, C. , COX, Adrienne, D. , DER, Channing, J.
发明人： GORDON, Leslie, B. , COLLINS, Francis, S. , GLOVER, Thomas , GLYNN, Michael, W. , CAPELL, Brian, C. , COX, Adrienne, D. , DER, Channing, J.
IPC分类号： A61K31/4704 , A61K31/473 , A61K31/445 , A61K31/255 , A61K31/221 , A61P43/00 , A61P9/10 , A61P21/00 , A61P25/00
摘要： Although it can he farnesylated, the mutant lamin A protein expressed in Hutchison Gilford Progeria Syndrome (HGPS) cannot be defarnesylated because the characteristic mutation causes deletion of a cleavage site necessary for binding the protease ZMPSTE24 and effecting defarnesylation. The result is an aberrant farnesylated protein (called "progerin") that alters normal lamin A function as a dominant negative, as well as assuming its own aberrant function through its association with the nuclear membrane. The retention of farnesylation, and potentially other abnormal properties of progerin and other abnormal lamin gene protein products, produces disease. Farnesyltransferase inhibitors (FTIs) (both direct effectors and indirect inhibitors) will inhibit the formation of progerin, cause a decrease in lamin A protein, and/or an increase prelamin A protein. Decreasing the amount of aberrant protein improves cellular effects caused by and progerin expression. Similarly, treatment with FTIs should improve disease status in progeria and other laminopathies. In addition, elements of atherosclerosis and aging in non- laminopathy individuals will improve after treatment with farnesyltransferase inhibitors.

2. 发明申请

WO2006081444A2 FARNESYLTRANSFERASE INHIBITORS FOR TREATMENT OF LAMINOPATHIES, CELLULAR AGING AND ATHEROSCLEROSIS 审中-公开
标题翻译：用于治疗肌肉瘤，细胞衰老和甲状腺功能亢进症的纤维素酶抑制剂
公开(公告)号：WO2006081444A2
公开(公告)日：2006-08-03
申请号：PCT/US2006002977
申请日：2006-01-27
申请人： CRETARY OF THE DEPT OF HEALTH , PROGERIA RES FOUNDATION , UNIV MICHIGAN , UNIV NORTH CAROLINA , GORDON LESLIE B , COLLINS FRANCIS S , GLOVER THOMAS , GLYNN MICHAEL W , CAPELL BRIAN C , COX ADRIENNE D , DER CHANNING J
发明人： GORDON LESLIE B , COLLINS FRANCIS S , GLOVER THOMAS , GLYNN MICHAEL W , CAPELL BRIAN C , COX ADRIENNE D , DER CHANNING J
CPC分类号： A61K31/255 , A61K31/221 , A61K31/445 , A61K31/4704 , A61K31/473
摘要： Although it can he farnesylated, the mutant lamin A protein expressed in Hutchison Gilford Progeria Syndrome (HGPS) cannot be defarnesylated because the characteristic mutation causes deletion of a cleavage site necessary for binding the protease ZMPSTE24 and effecting defarnesylation. The result is an aberrant farnesylated protein (called "progerin") that alters normal lamin A function as a dominant negative, as well as assuming its own aberrant function through its association with the nuclear membrane. The retention of farnesylation, and potentially other abnormal properties of progerin and other abnormal lamin gene protein products, produces disease. Farnesyltransferase inhibitors (FTIs) (both direct effectors and indirect inhibitors) will inhibit the formation of progerin, cause a decrease in lamin A protein, and/or an increase prelamin A protein. Decreasing the amount of aberrant protein improves cellular effects caused by and progerin expression. Similarly, treatment with FTIs should improve disease status in progeria and other laminopathies. In addition, elements of atherosclerosis and aging in non- laminopathy individuals will improve after treatment with farnesyltransferase inhibitors.
摘要翻译：虽然它可以进行法呢基化，但是在和记吉尔福德前列腺综合症（Hutchison Gilford Progeria Syndrome，HGPS）中表达的突变型蛋白A蛋白不能被脱甲基化，因为特征性突变导致缺失结合蛋白酶ZMPSTE24所必需的切割位点并进行脱甲酰化。结果是异常的法呢基蛋白（称为“progerin”），其改变正常的lamin A功能作为显性阴性，并且通过与核膜的结合来假设其自身的异常功能。法尼基化的保留以及progerin和其他异常的lamin基因蛋白产物的潜在的其他异常性质产生疾病。法尼基转移酶抑制剂（FTIs）（直接作用剂和间接抑制剂）将抑制progerin的形成，导致lamin A蛋白的降低和/或增加的prelamin A蛋白。减少异常蛋白质的量会改善由progerin表达引起的细胞效应。同样，FTIs治疗应改善progeria和其他层层病的疾病状况。此外，用法呢基转移酶抑制剂治疗后，动脉粥样硬化和非病变个体的老化成分将会改善。

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