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1. 发明申请

WO2012112674A2 COMPOUNDS AND METHODS OF USE THEREOF FOR TREATING NEURODEGENERATIVE DISORDERS 审中-公开
标题翻译：用于治疗神经损伤性疾病的化合物及其使用方法
公开(公告)号：WO2012112674A2
公开(公告)日：2012-08-23
申请号：PCT/US2012025228
申请日：2012-02-15
申请人： UNIV JOHNS HOPKINS , ZACK DONALD J , BERLINICKE CYNTHIA , HACKLER LASZLO , YANG ZHIYONG , STEINER JOSEPH P , VOJKOVSKY TOMAS , FERRARIS DANA , SLUSHER BARBARA S , INGLESE JAMES
发明人： ZACK DONALD J , BERLINICKE CYNTHIA , HACKLER LASZLO , YANG ZHIYONG , STEINER JOSEPH P , VOJKOVSKY TOMAS , FERRARIS DANA , SLUSHER BARBARA S , INGLESE JAMES
CPC分类号： C07D403/12 , C07D401/14 , C07D403/14 , C07D453/02
摘要： Compounds, compositions, kits and methods for treating conditions related to neurodegeneration or ocular disease, are disclosed.
摘要翻译：公开了用于治疗与神经变性或眼部疾病相关的病症的化合物，组合物，试剂盒和方法。

2. 发明申请

WO2010017541A2 COMPOSITIONS AND METHODS FOR TREATMENT OF NEURODEGENERATIVE DISEASE 审中-公开
标题翻译：用于治疗神经病变疾病的组合物和方法
公开(公告)号：WO2010017541A2
公开(公告)日：2010-02-11
申请号：PCT/US2009053241
申请日：2009-08-08
申请人： UNIV JOHNS HOPKINS , ZACK DONALD J , YANG ZHIYONG , BERLINICKE CYNTHIA , QUIGLEY HARRY
发明人： ZACK DONALD J , YANG ZHIYONG , BERLINICKE CYNTHIA , QUIGLEY HARRY
IPC分类号： A61K31/404 , A61P25/28 , A61P27/06
CPC分类号： A61K31/404 , A61K31/00 , A61K31/496 , A61K45/06 , A61K2300/00
摘要： Compounds, compositions, kits and methods for treating conditions related to neurodegeneration or ocular disease, are disclosed.
摘要翻译：公开了用于治疗与神经变性或眼部疾病相关的病症的化合物，组合物，试剂盒和方法。

3. 发明申请

WO2004007673A3 NEURONAL GENE EXPRESSION PATTERNS 审中-公开
标题翻译：神经元基因表达模式
公开(公告)号：WO2004007673A3
公开(公告)日：2004-11-18
申请号：PCT/US0321729
申请日：2003-07-14
申请人： UNIV JOHNS HOPKINS , ZACK DONALD J , KAGEYAMA MASAAKI
发明人： ZACK DONALD J , KAGEYAMA MASAAKI
IPC分类号： A61K38/00 , C07K14/47 , C12Q1/68 , A61K39/00 , A61K39/395 , C07K16/00 , C07K16/18 , C07K16/22 , C07K16/40
CPC分类号： C12Q1/6883 , A61K38/00 , A61K2039/505 , C07K14/47 , C12Q2600/158
摘要： Neuronal cell death, as modeled by removal of serum or NGF from growth medium, is characterized by many changes in gene expression. Gene expression was compared before and after withdrawal of serum or NGF. These results provide clues to underlying molecular processes occurring during neuronal and photoreceptor degeneration, and provide direction for future cell-based studies.
摘要翻译：如通过从生长培养基中去除血清或NGF来模拟的，神经元细胞死亡的特征在于基因表达的许多变化。比较血清或NGF撤出前后的基因表达。这些结果提供了神经元和光感受器退化过程中发生的潜在分子过程的线索，并为未来基于细胞的研究提供了方向。

4. 发明申请

WO2004007674A3 NEURONAL AND RETINAL GENE EXPRESSION PATTERNS 审中-公开
标题翻译：神经元和视网膜基因表达模式
公开(公告)号：WO2004007674A3
公开(公告)日：2004-10-07
申请号：PCT/US0321737
申请日：2003-07-14
申请人： UNIV JOHNS HOPKINS , ZACK DONALD J , HACKAM ABIGAIL SHOSHANNA
发明人： ZACK DONALD J , HACKAM ABIGAIL SHOSHANNA
IPC分类号： A61K38/00 , C07K14/47 , C12N5/08 , C12Q1/68 , A61K39/00 , A61K39/395 , C07K16/00 , C07K16/18 , C07K16/22 , C07K16/24
CPC分类号： C12Q1/6883 , A61K38/00 , A61K2039/505 , C07K14/47 , C12Q1/6837 , C12Q2600/158
摘要： The retinal degeneration (rdl) mutant mouse exhibits rapid rod photoreceptor degeneration caused by a mutation in the rod photoreceptor-specific gene cGMP phosphodiesterase beta (PDE). One intriguing aspect of the rdl phenotype is a secondary wave of cone photoreceptor death that follows loss of rods. In this study, we investigated gene expression changes associated with the progression of photoreceptor degeneration in rdl mice using a custom retina microarray. The microarray contains 5,376 DNA fragments that correspond to mouse genes known or postulated to be involved in normal retinal function, development, or disease. Gene expression in rdl retina was compared with age-matched wild-type controls at three time-points corresponding to critical stages in retina degeneration: peak of rod degeneration, early in cone degeneration and during cone degeneration. Statistical significance analyses demonstrated that approximately 3% of the genes on the microarray were differentially expressed, including known genes and genes that had not been previously implicated in degeneration. Interestingly, there was less overlap in the genes that were upregulated at each stage of degeneration, suggesting the involvement of distinct molecular pathways. Genes involved in transport, signalling and cytoskeleton were differentially expressed during rod degeneration whereas genes involved in growth and proliferation, oxidative stress and protein modification were increased prior to and during cone degeneration. These results provide clues to underlying molecular processes occurring during photoreceptor degeneration, and provide direction for future cell-based studies.
摘要翻译：视网膜变性（rdl）突变小鼠表现出由杆状光感受器特异性基因cGMP磷酸二酯酶β（PDE）中的突变引起的快速视杆光感受器变性。 rdl表型的一个有趣的方面是锥形光感受器死亡的次级波，其在棒损失之后。在这项研究中，我们使用定制视网膜微阵列研究了rdl小鼠中与光感受器变性进展相关的基因表达变化。微阵列含有5,376个DNA片段，其对应于已知或假定参与正常视网膜功能，发育或疾病的小鼠基因。将rdl视网膜中的基因表达与年龄匹配的野生型对照在对应于视网膜变性关键阶段的三个时间点进行比较：棒状变性的峰值，锥状变性早期和锥状变性期间。统计学显着性分析表明，微阵列上约3％的基因是差异表达的，包括已知的基因和先前未涉及退化的基因。有趣的是，在每个退化阶段上调的基因重叠较少，这表明不同分子途径的参与。涉及运输，信号传导和细胞骨架的基因在杆变性过程中差异表达，而参与生长和增殖，氧化应激和蛋白质修饰的基因在锥变性之前和期间增加。这些结果为光感受器退化过程中发生的潜在分子过程提供了线索，并为未来基于细胞的研究提供了方向。

5. 发明申请

WO2004007675A2 NEURONAL AND OPTIC NERVE GENE EXPRESSION PATTERNS 审中-公开
标题翻译：神经元和视神经基因表达模式
公开(公告)号：WO2004007675A2
公开(公告)日：2004-01-22
申请号：PCT/US0321738
申请日：2003-07-14
申请人： UNIV JOHNS HOPKINS , ZACK DONALD J , QUIGLEY HARRY A
发明人： ZACK DONALD J , QUIGLEY HARRY A
IPC分类号： A61K38/00 , C07K14/47 , C12Q1/68 , C12N
CPC分类号： C07K14/47 , A61K38/00 , A61K2039/505 , C12Q1/6883 , C12Q2600/158
摘要： The optic nerve axotomy model in mouse exhibits rapid changes in gene expression. Genes identified by microarray analysis as differentially expressed or modulated in this model, can be used diagnostically, therapeutically, and in drug discovery. These results provide clues to underlying molecular processes occurring during optic nerve degeneration, and provide direction for future cell-based studies.
摘要翻译：小鼠中的视神经轴突切断模型表现出基因表达的快速变化。通过微阵列分析鉴定出的差异表达或调节的基因可用于诊断，治疗和药物研发。这些结果提供了视神经退行期间发生的潜在分子过程的线索，并为未来基于细胞的研究提供了方向。

6. 发明申请

WO2016130460A2 PHTHALAZINONE PYRAZOLE DERIVATIVES FOR TREATING RETINAL DEGENERATIVE DISEASE 审中-公开
标题翻译：肼唑酮吡唑类衍生物治疗视网膜变性疾病
公开(公告)号：WO2016130460A2
公开(公告)日：2016-08-18
申请号：PCT/US2016016957
申请日：2016-02-08
申请人： UNIV JOHNS HOPKINS
发明人： ZACK DONALD J , FULLER JOHN A
IPC分类号： C07D403/12 , A61K31/501 , A61K31/502 , A61P25/00 , A61P25/28 , C07D237/26
CPC分类号： A61K31/502
摘要： Compounds, compositions, kits and methods for treating conditions related to Retinal degenerations, and related diseases, including retinitis pigmentosa and atrophic age-related macular degeneration, are disclosed.
摘要翻译：公开了用于治疗与视网膜变性有关的病症的化合物，组合物，试剂盒和方法以及相关疾病，包括色素性视网膜炎和萎缩性年龄相关性黄斑变性。

7. 发明申请

WO2016130460A3 PHTHALAZINONE PYRAZOLE DERIVATIVES FOR TREATING RETINAL DEGENERATIVE DISEASE 审中-公开
公开(公告)号：WO2016130460A3
公开(公告)日：2016-11-03
申请号：PCT/US2016016957
申请日：2016-02-08
申请人： UNIV JOHNS HOPKINS
发明人： ZACK DONALD J , FULLER JOHN A
IPC分类号： C07D403/12 , A61K31/501 , A61K31/502 , A61P25/00 , A61P25/28 , C07D237/26
CPC分类号： A61K31/502
摘要： Compounds, compositions, kits and methods for treating conditions related to Retinal degenerations, and related diseases, including retinitis pigmentosa and atrophic age-related macular degeneration, are disclosed.

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