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1. 发明申请

WO2003024409A3 Q4N2NEG2 ENHANCES CFTR ACTIVITY 审中-公开
公开(公告)号：WO2003024409A3
公开(公告)日：2003-03-27
申请号：PCT/US2002/030094
申请日：2002-09-23
申请人： CASE WESTERN RESERVE UNIVERSITY , DAVIS, Pamela, B. , MA, Jianjie , GERKEN, Thomas
发明人： DAVIS, Pamela, B. , MA, Jianjie , GERKEN, Thomas
IPC分类号： A61K38/17 , C07K14/435
摘要： Phosphorylation of the cystic fibrosis transmembrane conductance regulator (CFTR) by cyclic AMP- dependent protein kinase (PAK) is essential for opening the CFTR chloride channel. A short segment containing many negatively charged amino acids (817-838, NEG2) within the regulatory (R) domain of CFTR is a critical regulator of the chloride channel activity. An isolated NEG2 polypeptide may be expressed as a separate sequence that stimulates CFTR channel openings at lowe concentrations, but that inhibits CFTR channel openings at higher concentrations. Residues in the NEG2 sequence were substituted to produce a polypeptide that exerts only an activating effect on CFTR. One such polypeptide is the Q4N2NEG2 polypeptide. Exogenous Q4N2NEG2 exerts stimulatory effects on both wild-type and mutant G551D CFTR function, without exhibiting inhibitory activity at any concentration.

2. 发明申请

WO2003024409A2 Q4N2NEG2 ENHANCES CFTR ACTIVITY 审中-公开
标题翻译： Q4N2NEG2增强CFTR活性
公开(公告)号：WO2003024409A2
公开(公告)日：2003-03-27
申请号：PCT/US2002/030094
申请日：2002-09-23
申请人： CASE WESTERN RESERVE UNIVERSITY , DAVIS, Pamela, B. , MA, Jianjie , GERKEN, Thomas
发明人： DAVIS, Pamela, B. , MA, Jianjie , GERKEN, Thomas
IPC分类号： A61K
CPC分类号： C07K14/4712 , A61K38/00
摘要： Phosphorylation of the cystic fibrosis transmembrane conductance regulator (CFTR) by cyclic AMP- dependent protein kinase (PAK) is essential for opening the CFTR chloride channel. A short segment containing many negatively charged amino acids (817-838, NEG2) within the regulatory (R) domain of CFTR is a critical regulator of the chloride channel activity. An isolated NEG2 polypeptide may be expressed as a separate sequence that stimulates CFTR channel openings at lowe concentrations, but that inhibits CFTR channel openings at higher concentrations. Residues in the NEG2 sequence were substituted to produce a polypeptide that exerts only an activating effect on CFTR. One such polypeptide is the Q4N2NEG2 polypeptide. Exogenous Q4N2NEG2 exerts stimulatory effects on both wild-type and mutant G551D CFTR function, without exhibiting inhibitory activity at any concentration.
摘要翻译：通过环AMP-依赖性蛋白激酶（PAK）的囊性纤维化跨膜电导调节因子（CFTR）的磷酸化对于打开CFTR氯离子通道是必不可少的。在CFTR的调节（R）域内含有许多带负电荷的氨基酸（817-838，NEG2）的短链段是氯通道活性的关键调节剂。分离的NEG2多肽可以表达为在低浓度刺激CFTR通道开口的单独序列，但是在较高浓度下抑制CFTR通道开口。取代NEG2序列中的残基以产生对CFTR仅具有活化作用的多肽。一种这样的多肽是Q4N2NEG2多肽。外源性Q4N2NEG2对野生型和突变型G551D CFTR功能均有刺激作用，不影响任何浓度的抑制活性。

3. 发明申请

WO2006132928A3 METHODS AND COMPOSITIONS FOR TREATING INFLAMMATION 审中-公开
公开(公告)号：WO2006132928A3
公开(公告)日：2006-12-14
申请号：PCT/US2006/021304
申请日：2006-06-02
申请人： CASE WESTERN RESERVE UNIVERSITY , DAVIS, Pamela, B.
发明人： DAVIS, Pamela, B.
IPC分类号： A01N37/10
摘要： A method of treating a subject with a cystic fibrosis related disorder includes administering a therapeutically effective amount of at least one PPARγ agonist or a derivative thereof.

4. 发明申请

WO1997046100A1 SERPIN ENZYME COMPLEX RECEPTOR-MEDIATED GENE TRANSFER 审中-公开
标题翻译： SERPIN ENZYME复合受体媒介基因转移
公开(公告)号：WO1997046100A1
公开(公告)日：1997-12-11
申请号：PCT/US1997009858
申请日：1997-06-03
申请人： CASE WESTERN RESERVE UNIVERSITY
发明人： CASE WESTERN RESERVE UNIVERSITY , FERKOL, Thomas, W., Jr. , DAVIS, Pamela, B. , ZIADY, Assem-Galal
IPC分类号： A01N43/04
CPC分类号： C12Y304/21022 , A61K47/645 , A61K47/6807 , A61K47/6811 , A61K47/6849 , A61K48/00 , C07H21/00 , C07K14/705 , C07K16/18 , C07K19/00 , C07K2319/00 , C12N9/644 , C12N15/87
摘要： Nucleic acids are compacted, substantially without aggregation, to facilitate their uptake by target cells of an organism to which the compacted material is administered. The nucleic acids may achieve a clinical effect as a result of gene expression, hybridization to endogenous nucleic acids whose expression is undesired, or site-specific integration so that a target gene is replaced, modified or deleted. The targeting may be enhanced by means of a target cell-binding moiety. The nucleic acid is preferably compacted to a condensed state.
摘要翻译：核酸被压实，基本上没有聚集，以促进其被施用压实材料的生物体的靶细胞摄取。核酸可以作为基因表达的结果获得临床效果，与表达不期望的内源性核酸的杂交或位点特异性整合以使靶基因被替换，修饰或缺失。靶向可以通过靶细胞结合部分增强。优选将核酸压缩至冷凝状态。

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