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1. 发明申请

WO2012129084A3 GLUCOSYLCERAMIDE SYNTHASE INHIBITORS 审中-公开
公开(公告)号：WO2012129084A3
公开(公告)日：2012-09-27
申请号：PCT/US2012/029417
申请日：2012-03-16
申请人： GENZYME CORPORATION , BOURQUE, Elyse , CELATKA, Cassandra , HIRTH, Bradford , METZ, Markus , ZHAO, Zhong , SKERLJ, Renato , XIANG, Yibin , JANCISICS, Katherine , MARSHALL, John , CHENG, Seng , SCHEULE, Ronald , CABRERA-SALAZAR, Mario , GOOD, Andrew
发明人： BOURQUE, Elyse , CELATKA, Cassandra , HIRTH, Bradford , METZ, Markus , ZHAO, Zhong , SKERLJ, Renato , XIANG, Yibin , JANCISICS, Katherine , MARSHALL, John , CHENG, Seng , SCHEULE, Ronald , CABRERA-SALAZAR, Mario , GOOD, Andrew
IPC分类号： A01N43/00
摘要： The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.

2. 发明申请

WO2006053043A2 METHODS OF TREATING DIABETES MELLITUS 审中-公开
标题翻译：治疗糖尿病的方法
公开(公告)号：WO2006053043A2
公开(公告)日：2006-05-18
申请号：PCT/US2005/040596
申请日：2005-11-09
申请人： GENZYME CORPORATION , CHENG, Seng, H. , YEW, Nelson, S. , SCHEULE, Ronald, K. , ZHAO, Hongmei
发明人： CHENG, Seng, H. , YEW, Nelson, S. , SCHEULE, Ronald, K. , ZHAO, Hongmei
IPC分类号： A61K31/395 , A61K31/4025 , A61P3/10
CPC分类号： A61K31/4025 , A61K31/395 , A61K31/40 , Y10S514/866
摘要： The invention provides methods of treating a diabetic subject comprising administering a glucosylceramide synthase inhibitor to the subject.
摘要翻译：本发明提供了治疗糖尿病受试者的方法，包括向受试者施用葡糖基神经酰胺合酶抑制剂。

3. 发明申请

WO2012129084A2 GLUCOSYLCERAMIDE SYNTHASE INHIBITORS 审中-公开
标题翻译： GLUCOSYLCERAMIDE SYNTHASE抑制剂
公开(公告)号：WO2012129084A2
公开(公告)日：2012-09-27
申请号：PCT/US2012029417
申请日：2012-03-16
申请人： GENZYME CORP , BOURQUE ELYSE , CELATKA CASSANDRA , HIRTH BRADFORD , METZ MARKUS , ZHAO ZHONG , SKERLJ RENATO , XIANG YIBIN , JANCISICS KATHERINE , MARSHALL JOHN , CHENG SENG , SCHEULE RONALD , CABRERA-SALAZAR MARIO , GOOD ANDREW
发明人： BOURQUE ELYSE , CELATKA CASSANDRA , HIRTH BRADFORD , METZ MARKUS , ZHAO ZHONG , SKERLJ RENATO , XIANG YIBIN , JANCISICS KATHERINE , MARSHALL JOHN , CHENG SENG , SCHEULE RONALD , CABRERA-SALAZAR MARIO , GOOD ANDREW
IPC分类号： A61K31/55
CPC分类号： C07D487/08 , A61K31/439 , A61K31/454 , A61K31/46 , A61K31/506 , A61K31/551 , A61K38/47 , C07D453/00 , C07D453/02 , C07D455/02 , C12Y302/01022
摘要： The invention relates to inhibitors of glucosylceramide synthase (GCS) useful for the treatment metabolic diseases, such as lysosomal storage diseases, either alone or in combination with enzyme replacement therapy, and for the treatment of cancer.
摘要翻译：本发明涉及可用于单独或与酶替代疗法组合治疗代谢性疾病如溶酶体贮积病的葡萄糖神经酰胺合成酶（GCS）抑制剂和癌症治疗。

4. 发明申请

WO2007084743A3 INTRAVENTRICULAR PROTEIN DELIVERY FOR AMYOTROPHIC LATERAL SCLEROSIS 审中-公开
标题翻译：宫内膜间质性输卵管造影
公开(公告)号：WO2007084743A3
公开(公告)日：2008-11-27
申请号：PCT/US2007001599
申请日：2007-01-22
申请人： GENZYME CORP , DODGE JAMES , SCHEULE RONALD
发明人： DODGE JAMES , SCHEULE RONALD
IPC分类号： A61K38/00 , A61K49/00 , C07K14/00
CPC分类号： A61K38/30
摘要： Amyotrophic Lateral Sclerosis can be successfully treated using intraventricular delivery of a neurotrophic growth factor, IGF-I . The administration can be performed slowly to achieve maximum effect. Effects are seen on both sides of the blood-brain barrier, making this a delivery means for Amyotrophic Lateral Sclerosis which affects both brain and skeletal muscle.
摘要翻译：肌营养不良性侧索硬化可以使用脑室内输送神经营养生长因子IGF-I来成功治疗。可以缓慢执行给药以达到最大效果。在血脑屏障的两侧看到效果，使其成为影响脑和骨骼肌的肌萎缩性侧索硬化的递送方式。

5. 发明申请

WO2006060641A2 METHODS FOR TARGETED DELIVERY OF GENETIC MATERIAL TO THE LIVER 审中-公开
标题翻译：靶向向肝输送遗传物质的方法
公开(公告)号：WO2006060641A2
公开(公告)日：2006-06-08
申请号：PCT/US2005043590
申请日：2005-12-01
申请人： GENZYME CORP , SCHEULE RONALD K , HODGES BRADLEY L
发明人： SCHEULE RONALD K , HODGES BRADLEY L
IPC分类号： A61K48/00 , A61F2/958
CPC分类号： A61K48/0075
摘要： The present invention provides methods for enhanced delivery of various therapeutic agents, such as gene therapy agents, to the vasculature of a target organ in a mammalian subject. The methods for targeted gene therapy in the mammalian liver as a whole, or in a single hepatic lobe, are disclosed. The disclosed methods rely on minimally invasive catheter-based procedures wherein a target organ is isolated and treated locally with a gene therapy agent. The methods offer more efficient and localized transfection of tissue and are wellsuited for gene therapy in human subjects.
摘要翻译：本发明提供了用于将各种治疗剂（例如基因治疗剂）增强递送至哺乳动物受试者中靶器官的脉管系统的方法。公开了在整个哺乳动物肝脏中或在单个肝叶中靶向基因治疗的方法。所公开的方法依赖于微创的基于导管的手术，其中目标器官被分离并且用基因治疗剂局部治疗。这些方法提供更有效和局部的组织转染，并适用于人类受试者的基因治疗。

6. 发明申请

WO2008137490A3 SYSTEMIC INSULIN-LIKE GROWTH FACTOR-1 THERAPY REDUCES DIABETIC PERIPHERAL NEUROPATHY AND IMPROVES RENAL FUNCTION IN DIABETIC NEPHROPATHY 审中-公开
公开(公告)号：WO2008137490A3
公开(公告)日：2008-11-13
申请号：PCT/US2008/062129
申请日：2008-05-01
申请人： GENZYME CORPORATION , CHU, Qiuming , SCHEULE, Ronald, K.
发明人： CHU, Qiuming , SCHEULE, Ronald, K.
IPC分类号： A61K31/426 , A61K31/522
摘要： The present invention provides methods of treatment of patients suffering from the complication of blood sugar disorders diabetic peripheral neuropathy and diabetic nephropathy by administration of IGF-1 via protein therapy or gene therapy It relates to methods of treating an individual having a diabetic disorder or a hyperglycemic disorder, compπsing administeπng to the individual an effective amount of a DNA vector expressing IGF-1Eb or IGF-1Ec in vivo or an effective amount of the IGF-1Eb or IGF-1Ec protein in the early hyperalgesia stage or in patient that have advanced to the hyposensitivity stage

7. 发明申请

WO2004001049A1 METHODS OF DELIVERING GENE THERAPY AGENTS 审中-公开
标题翻译：递送基因治疗药物的方法
公开(公告)号：WO2004001049A1
公开(公告)日：2003-12-31
申请号：PCT/US2003/019639
申请日：2003-06-24
申请人： GENZYME CORPORATION , BASKIN, Kevin, M. , EASTMAN, Simon, J. , SCHEULE, Ronald, K.
发明人： BASKIN, Kevin, M. , EASTMAN, Simon, J. , SCHEULE, Ronald, K.
IPC分类号： C12N15/63
CPC分类号： A61K48/0075 , A61K48/0083
摘要： The present invention provides methods for pressure-enhanced delivery of various therapeutic agents, such as gene therapy agents, to the vasculature of a target organ in a mammalian subject. Methods for targeted gene therapy in the mammalian liver as a whole, or in a single hepatic lobe, are disclosed. The disclosed methods rely on minimally invasive catheterbased procedures wherein a target organ is isolated and treated locally with a gene therapy agent. The methods offer more efficient and localized transfection of tissue and are well suited for gene therapy in human subjects.
摘要翻译：本发明提供了将各种治疗剂如基因治疗剂递送到哺乳动物受试者的靶器官的脉管系统的方法。公开了在哺乳动物肝脏中作为整体或在单个肝叶中靶向基因治疗的方法。所公开的方法依赖于基于微创的基于导管的程序，其中目标器官被分离并用基因治疗剂局部治疗。该方法提供更有效和局部的组织转染，非常适合人类受试者的基因治疗。

8. 发明申请

WO2008137490A2 SYSTEMIC INSULIN-LIKE GROWTH FACTOR-1 THERAPY REDUCES DIABETIC PERIPHERAL NEUROPATHY AND IMPROVES RENAL FUNCTION IN DIABETIC NEPHROPATHY 审中-公开
标题翻译：系统胰岛素样生长因子-1治疗可减少糖尿病性外周神经病变，改善糖尿病患者的肾功能
公开(公告)号：WO2008137490A2
公开(公告)日：2008-11-13
申请号：PCT/US2008062129
申请日：2008-05-01
申请人： GENZYME CORP , CHU QIUMING , SCHEULE RONALD K
发明人： CHU QIUMING , SCHEULE RONALD K
IPC分类号： A61K48/00
CPC分类号： A61K38/30 , A61K48/00 , C12N15/86 , C12N2750/14143 , C12N2830/008
摘要： The present invention provides methods of treatment of patients suffering from the complications of blood sugar disorders: diabetic peripheral neuropathy and diabetic nephropathy by administration of IGF-1 via protein therapy or gene therapy. It relates to methods of treating an individual having a diabetic disorder or a hyperglycemic disorder, comprising administering to the individual an effective amount of a DNA vector expressing IGF-I Eb or IGF-I Ec in vivo or an effective amount of at the IGF-I Eb or IGF-I Ec protein in the early hyperalgesia stage or in patients that have advanced to the hyposensitivity stage. Treatment at the early hyperalgesia stage prevents subsequent hyposensitivity with increases or maintenance of sensory nerve function. IGF-I Eb or IGF-I Ec treatment also increases muscle mass and improves overall mobility, which indicates a treatment-related improvement in motor function. Treatment with IGF-I Eb or IGF-I Ec at the hyposensitivity stage reverses hyposensitivity and improves muscle mass and overall health. Systemic IGF- 1 provides a therapeutic modality for treating hyposensitivity associated with DPN. In addition, IGF-I Eb or IGF-I Ec provides a therapeutic modality for treating diabetic nephropathy. IGF-I Eb or IGF-I Ec improves renal function as evidenced by a modulation in serum albumin concentration and a reduction in urine volume and protein levels. IGF-I Eb or IGF-1 Ec also reduces diabetic glomerulosclerosis.
摘要翻译：本发明提供了通过蛋白质治疗或基因治疗施用IGF-1来治疗患有血糖紊乱并发症的患者的糖尿病性周围神经病变和糖尿病性肾病的方法。本发明涉及治疗患有糖尿病或高血糖病症的个体的方法，包括向个体施用有效量的体内表达IGF-I Eb或IGF-1 Ec的DNA载体或有效量的IGF- I Eb或IGF-I Ec蛋白在早期痛觉过敏阶段或患有进展至低敏感期的患者。早期痛觉过敏阶段的治疗可通过增加或维持感觉神经功能阻止随后的敏感性。 IGF-I Eb或IGF-I Ec治疗也增加肌肉质量并改善整体移动性，这表明运动功能的治疗相关改善。 IGF-I Eb或IGF-I的治疗在低敏感期反转低敏感性，改善肌肉质量和整体健康。系统性IGF-1提供治疗与DPN相关的敏感性的治疗方式。此外，IGF-I Eb或IGF-I Ec提供治疗糖尿病性肾病的治疗方法。 IGF-I Eb或IGF-I Ec改善肾功能，如通过血清白蛋白浓度的调节和尿量和蛋白质水平的降低所证明的。 IGF-I Eb或IGF-1 Ec还可减少糖尿病肾小球硬化。

9. 发明申请

WO2007084743A2 INTRAVENTRICULAR PROTEIN DELIVERY FOR AMYOTROPHIC LATERAL SCLEROSIS 审中-公开
标题翻译：宫内膜性交感神经损伤
公开(公告)号：WO2007084743A2
公开(公告)日：2007-07-26
申请号：PCT/US2007/001599
申请日：2007-01-22
申请人： GENZYME CORPORATION , DODGE, James , SCHEULE, Ronald
发明人： DODGE, James , SCHEULE, Ronald
IPC分类号： A61K38/30
CPC分类号： A61K38/30
摘要： Amyotrophic Lateral Sclerosis can be successfully treated using intraventricular delivery of a neurotrophic growth factor, IGF-I . The administration can be performed slowly to achieve maximum effect. Effects are seen on both sides of the blood-brain barrier, making this a delivery means for Amyotrophic Lateral Sclerosis which affects both brain and skeletal muscle.
摘要翻译：可以使用脑室内递送神经营养生长因子IGF-I来成功地治疗肌萎缩性侧索硬化症。可以缓慢执行给药以达到最大效果。在血脑屏障的两侧看到效果，使其成为影响脑和骨骼肌的肌萎缩性侧索硬化的递送手段。

10. 发明申请

WO2006053043A3 METHODS OF TREATING DIABETES MELLITUS 审中-公开
标题翻译：治疗糖尿病的方法
公开(公告)号：WO2006053043A3
公开(公告)日：2006-08-24
申请号：PCT/US2005040596
申请日：2005-11-09
申请人： GENZYME CORP , CHENG SENG H , YEW NELSON S , SCHEULE RONALD K , ZHAO HONGMEI
发明人： CHENG SENG H , YEW NELSON S , SCHEULE RONALD K , ZHAO HONGMEI
IPC分类号： A61K31/395 , A61K31/4025 , A61P3/10
CPC分类号： A61K31/4025 , A61K31/395 , A61K31/40 , Y10S514/866
摘要： The invention provides methods of treating a diabetic subject comprising administering a glucosylceramide synthase inhibitor to the subject.
摘要翻译：本发明提供治疗糖尿病受试者的方法，包括向受试者施用葡萄糖神经酰胺合酶抑制剂。

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