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1. 发明申请

WO2006116301A1 NEUROPROTECTIVE COMPOUNDS FOR TREATING OPTIC NEUROPATHIES 审中-公开
标题翻译：用于治疗光神经病变的神经保护性化合物
公开(公告)号：WO2006116301A1
公开(公告)日：2006-11-02
申请号：PCT/US2006/015457
申请日：2006-04-24
申请人： THE JOHNS HOPKINS UNIVERSITY , KERRISON, John, Barnwell , ZACK, Donald, J.
发明人： KERRISON, John, Barnwell , ZACK, Donald, J.
IPC分类号： C07D211/56
CPC分类号： C07D239/50 , C07C233/25 , C07C239/16 , C07C251/86 , C07C311/46 , C07C2602/20 , C07D207/416 , C07D333/22 , C07D417/12 , G01N33/5058 , G01N33/54326 , G01N33/56966 , G01N2800/164
摘要： Neuroprotective compounds for treating optic neuropathies and screening methods for identifying neuroprotective compounds.
摘要翻译：用于治疗视神经病的神经保护性化合物和用于鉴定神经保护性化合物的筛选方法。

2. 发明申请

WO2004007673A2 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：WO2004007673A2
公开(公告)日：2004-01-22
申请号：PCT/US2003/021729
申请日：2003-07-14
申请人： THE JOHNS HOPKINS UNIVERSITY , ZACK, Donald, J. , KAGEYAMA, Masaaki
发明人： ZACK, Donald, J. , KAGEYAMA, Masaaki
IPC分类号： C12N
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：通过从生长培养基中去除血清或NGF来建模的神经元细胞死亡的特征在于基因表达的许多变化。在血清或NGF撤出前后比较基因表达。这些结果提供了在神经元和感光器变性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。

3. 发明申请

WO2004007675A3 NEURONAL AND OPTIC NERVE GENE EXPRESSION PATTERNS 审中-公开
公开(公告)号：WO2004007675A3
公开(公告)日：2004-01-22
申请号：PCT/US2003/021738
申请日：2003-07-14
申请人： THE JOHNS HOPKINS UNIVERSITY , ZACK, Donald, J. , QUIGLEY, Harry, A.
发明人： ZACK, Donald, J. , QUIGLEY, Harry, A.
IPC分类号： A61K39/00
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.

4. 发明申请

WO2010017541A3 COMPOSITIONS AND METHODS FOR TREATMENT OF NEURODEGENERATIVE DISEASE 审中-公开
公开(公告)号：WO2010017541A3
公开(公告)日：2010-02-11
申请号：PCT/US2009/053241
申请日：2009-08-08
申请人： THE JOHNS HOPKINS UNIVERSITY , 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 , A61P27/06 , A61P25/28
摘要： Compounds, compositions, kits and methods for treating conditions related to neurodegeneration or ocular disease, are disclosed.

5. 发明申请

WO2004007674A2 NEURONAL AND RETINAL GENE EXPRESSION PATTERNS 审中-公开
标题翻译：神经和基因表达模式
公开(公告)号：WO2004007674A2
公开(公告)日：2004-01-22
申请号：PCT/US2003/021737
申请日：2003-07-14
申请人： THE JOHNS HOPKINS UNIVERSITY , ZACK, Donald, J. , HACKAM, Abigail, Shoshanna
发明人： ZACK, Donald, J. , HACKAM, Abigail, Shoshanna
IPC分类号： C12N
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 β (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感光器特异性基因cGMP磷酸二酯酶β（PDE）中的突变引起的快速棒状光感受器变性。 rdl表型的一个有趣的方面是锥形光感受器死亡的次级波，其伴随着棒的损失。在这项研究中，我们使用定制的视网膜微阵列调查与rdl小鼠光感受器退化进展相关的基因表达变化。微阵列包含5,376个DNA片段，其对应于已知或假设参与正常视网膜功能，发育或疾病的小鼠基因。将视网膜中的基因表达与对应于视网膜变性的关键阶段的三个时间点与年龄匹配的野生型对照进行比较：杆变性的峰值，锥形变性早期和锥形变性。统计学意义分析表明，微阵列中约3％的基因差异表达，包括已知的基因和以前没有参与变性的基因。有趣的是，在每个退化阶段上调基因的重叠较少，表明不同分子途径的参与。参与转运，信号转导和细胞骨架的基因在杆变性过程中有差异表达，而在锥形变性之前和期间，参与生长和增殖，氧化应激和蛋白质修饰的基因都增加。这些结果提供了在光感受器变性过程中发生的潜在分子过程的线索，为未来基于细胞的研究提供了方向。

6. 发明公开

EP4323535A1 PROMOTERS FOR VIRAL-BASED GENE THERAPY 审中-实审
公开(公告)号：EP4323535A1
公开(公告)日：2024-02-21
申请号：EP22788957.3
申请日：2022-04-14
申请人： The Regents of the University of California , The Johns Hopkins University
发明人： WELSBIE, Derek , ZHANG, Pingwu , PATEL, Amit , LU, Tianlun , ZACK, Donald, J. , CHAU, Natalie
IPC分类号： C12N15/86 , A61K48/00

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