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1. 发明申请

US20060014695A1 Hpma-polyamine conjugates and uses therefore 审中-公开
标题翻译：因此，Hpma-聚胺共轭物和用途
公开(公告)号：US20060014695A1
公开(公告)日：2006-01-19
申请号：US10502985
申请日：2003-01-31
申请人： Hamidreza Ghandehari , Martin Woodle , Puthupparampil Scaria , Anjan Nan
发明人： Hamidreza Ghandehari , Martin Woodle , Puthupparampil Scaria , Anjan Nan
IPC分类号： A61K48/00 , A61K39/395
CPC分类号： C07K5/021 , A61K31/74 , A61K47/58 , C07K5/1008 , C07K5/1027 , C12N15/87 , C12N2810/405
摘要： The inventions provide compositions and methods for nucleic acid delivery comprising IIPMA conjugated to a polyamine. These compositions have the benefit of the steric hindrance of HPMA and the nucleic acid binding capability of a polyamine. Useful polyamines for this purpose include spermine, spermidine and their analogues, and DFMO. These polyamines have the ability not only to bind nucleic acids, but also have anti-cancer effects themselves. The compounds provided can also include ligand binding domains, such as vascular endothelial growth factors, somatostatin and somatostatin analogs, transferring, melanotropin, ApoE and ApoE peptides, von Willebrand's factor and von Willebrand's factor peptides, adenoviral fiber protein and adenoviral fiber protein peptides, PD 1 and PD 1 peptides, EGF and EGF peptides, RGD peptides, CCK peptides, antibody and antibody fragments, folate, pyridoxyl and sialyl-LewisX and chemical analogs. Methods for using these compositions to achieve a therapeutic effect, including for vaccination, are also provided.
摘要翻译：本发明提供了包含与多胺缀合的IIPMA的核酸递送的组合物和方法。这些组合物具有HPMA的空间位阻和多胺的核酸结合能力的益处。有用的多胺用于此目的包括精胺，亚精胺及其类似物，以及DFMO。这些多胺不仅具有结合核酸的能力，而且还具有抗癌作用。所提供的化合物还可以包括配体结合域，例如血管内皮生长因子，生长抑素和生长抑素类似物，转移，黑素素，ApoE和ApoE肽，血管性血友病因子和血管性血友病因子肽，腺病毒纤维蛋白和腺病毒纤维蛋白肽，PD 1和PD 1肽，EGF和EGF肽，RGD肽，CCK肽，抗体和抗体片段，叶酸，吡哆醛和唾液酸-LXXX和化学类似物。还提供了使用这些组合物来实现治疗效果的方法，包括用于疫苗接种。

2. 发明申请

US20060211637A1 Methods of down regulating target gene expression in vivo by introduction of interfering rna 审中-公开
标题翻译：通过引入干扰rna在体内下调靶基因表达的方法
公开(公告)号：US20060211637A1
公开(公告)日：2006-09-21
申请号：US10523714
申请日：2003-08-06
申请人： Puthupparampil Scaria , Martin Woodle , Patrick Lu , Qingquan Tang , Jun Xu , Frank Xie
发明人： Puthupparampil Scaria , Martin Woodle , Patrick Lu , Qingquan Tang , Jun Xu , Frank Xie
IPC分类号： A61K48/00 , C12N15/87 , C08L89/00
CPC分类号： A61K48/00 , C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/351 , C12N2310/3513 , C12N2310/53 , C12N2320/32
摘要： Methods and compositions are provided for down regulation of target gene expression in vivo by RNA interference. The methods are useful for target discovery and validation of gene-based drug development, and for treatment of human diseases.
摘要翻译：提供了通过RNA干扰在体内下调靶基因表达的方法和组合物。该方法可用于基于基因的药物开发和人类疾病治疗的靶发现和验证。

3. 发明申请

US20070237718A1 Multi-disciplinary approach to validating or indentifying targets using an in vivo system 审中-公开
标题翻译：使用体内系统验证或识别目标的多学科方法
公开(公告)号：US20070237718A1
公开(公告)日：2007-10-11
申请号：US11717603
申请日：2007-03-12
申请人： Patrick Lu , Puthupparampil Scaria , Martin Woodle
发明人： Patrick Lu , Puthupparampil Scaria , Martin Woodle
IPC分类号： G01N33/48 , C12Q1/00 , C12Q1/68
CPC分类号： G01N33/6803 , C12Q1/6886 , G01N33/5088 , G01N33/574
摘要： The present invention embodies a multi-disciplinary approach to validate or identify targets involved in any given biological process or pathway, such as an immune response, or progression or regression of disease. By introducing a target(s) with, e.g., gene delivery vector(s) or other foreign substance(s) to an in vivo system and by integrating, for example, pathological, pharmacological, bioassay, microarray and bioinformatics data obtained from the in vivo system, the present inventors are able to (1) identify one or more targets, e.g., genes, that are involved in a biological pathway of interest, (2) implement these identified targets for further analysis of the biological process or pathway and (3) provide a scalable approach in vivo for potential large quantities of target(s) discovery and validation. This process can be used in any number of applications, including the identification of agonists and antagonists to a biological process or pathway, which can lead to drugs and vaccine discovery.
摘要翻译：本发明体现了一种多学科方法来验证或鉴定涉及任何给定生物过程或途径（例如免疫应答或疾病进展或消退）的靶标。通过将诸如基因递送载体或其他外来物质的靶引入体内系统，并通过整合例如病理学，药理学，生物测定，微阵列和生物信息学数据，从本发明人能够（1）识别涉及生物学途径的一个或多个目标，例如基因，（2）实施这些鉴定的目标以进一步分析生物过程或途径和（ 3）为潜在的大量目标发现和验证在体内提供可扩展的方法。该方法可用于任何数量的应用，包括鉴定可导致药物和疫苗发现的生物过程或途径的激动剂和拮抗剂。

4. 发明申请

US20070219118A1 Therapeutic methods for nucleic acid delivery vehicles 审中-公开
标题翻译：核酸递送载体的治疗方法
公开(公告)号：US20070219118A1
公开(公告)日：2007-09-20
申请号：US11591611
申请日：2006-11-02
申请人： Patrick Lu , Puthupparampil Scaria , Martin Woodle , Frank Xie , Jun Xu , Quinn Tang
发明人： Patrick Lu , Puthupparampil Scaria , Martin Woodle , Frank Xie , Jun Xu , Quinn Tang
IPC分类号： A61K31/7052 , A61K38/00
CPC分类号： A61K48/0041 , A61K48/0008 , A61K48/0083 , A61K48/0091
摘要： It has been found that certain synthetic vectors and nucleic acid sequences that encode viral genomic sequences can, for example, be administered to a subject repeatedly as a vehicle for effectively delivering one or more therapeutic nucleic acid molecules or polypeptides to a cell or tissue. Accordingly, the disclosed nucleic acid delivery vehicles can be used, for instance, as part of a therapeutic regimen that involves an ongoing use of a therapeutic nucleic acid molecule or polypeptide.
摘要翻译：已经发现，编码病毒基因组序列的某些合成载体和核酸序列可以例如作为用于有效地将一种或多种治疗性核酸分子或多肽有效递送至细胞或组织的载体重复施用于受试者。因此，所公开的核酸递送载体可用于例如涉及正在使用治疗性核酸分子或多肽的治疗方案的一部分。

5. 发明申请

US20110312877A1 ENGINEERED TUNABLE NANOPARTICLES FOR DELIVERY OF THERAPEUTICS, DIAGNOSTICS, AND EXPERIMENTAL COMPOUNDS AND RELATED COMPOSITIONS FOR THERAPEUTIC USE 审中-公开
标题翻译：用于输送治疗药物，诊断和实验化合物的工程应用纳米颗粒及治疗用途的相关组合物
公开(公告)号：US20110312877A1
公开(公告)日：2011-12-22
申请号：US12919703
申请日：2009-02-26
申请人： Mark Berninger , Puthupparampil Scaria , Martin Woodle
发明人： Mark Berninger , Puthupparampil Scaria , Martin Woodle
IPC分类号： A61K38/16 , A61P31/10 , C07K19/00 , B82Y5/00
CPC分类号： B82Y5/00 , A61K39/07 , A61K39/39 , A61K47/60 , A61K47/641 , A61K47/6911 , A61K2039/55555 , A61K2039/6031 , C07K2317/21 , C07K2317/52
摘要： Biomedical nanoparticles are disclosed based on new engineered modular carrier macromolecules, on engineered macromolecules or associated entities providing an internal nanoparticle structure, and compositions for minimizing non-specific binding of the nanoparticles while enabling efficient and convenient targeting to cells and tissues. These nanoparticles may be used to deliver atomic or molecular or associated entities which are useful for diagnostics, primarily in vivo imaging, for therapeutics, for vaccines, or for experimental research. Nanoparticles comprising combinations of active entities such as gene inhibitors with gene expression cassettes or imaging agents with therapeutic agents, and polyamide compounds useful for treatment of microbial infections are also disclosed.
摘要翻译：公开了基于新的工程化模块化载体大分子，工程化大分子或提供内部纳米颗粒结构的相关实体的生物医学纳米颗粒，以及用于最小化纳米粒子的非特异性结合的组合物，同时能够有效和方便地靶向细胞和组织。这些纳米颗粒可用于递送原子或分子或相关实体，其可用于诊断，主要是体内成像，治疗剂，疫苗或实验研究。还公开了包含活性实体如基因抑制剂与基因表达盒或成像剂与治疗剂的组合的纳米颗粒，以及可用于治疗微生物感染的聚酰胺化合物。

6. 发明申请

US20060051315A1 Polymers for delivering peptides and small molecules in vivo 审中-公开
标题翻译：用于在体内递送肽和小分子的聚合物
公开(公告)号：US20060051315A1
公开(公告)日：2006-03-09
申请号：US10502986
申请日：2003-01-31
申请人： Puthupparampil Scaria , Martin Woodle
发明人： Puthupparampil Scaria , Martin Woodle
IPC分类号： A61K38/17 , C08G63/91
CPC分类号： C08G63/08 , A61K31/74 , A61K47/64 , C08G63/912 , C08G65/329 , C08G73/0233 , C08L2203/02
摘要： Certain hydrophilic polymers, such as a polyoxazoline, when conjugated to a polypeptide or small molecule agent, can enhance the bioavailability of the agent when administered in vivo. Accordingly, hydrophilic polymers of the invention can be used as a delivery vehicle to treat any number of disorders and/or confer a myriad of therapeutic benefits to a subject.
摘要翻译：当与多肽或小分子试剂缀合时，某些亲水性聚合物例如聚恶唑啉可以在体内施用时增强试剂的生物利用度。因此，本发明的亲水性聚合物可用作递送载体以治疗任何数量的病症和/或赋予受试者无数的治疗益处。

7. 发明申请

US20100028848A1 Compositions and Methods of Using siRNA to Knockdown Gene Expression and to Improve Solid Organ and Cell Transplantation 审中-公开
标题翻译：使用siRNA敲除基因表达和改善固体器官和细胞移植的组合物和方法
公开(公告)号：US20100028848A1
公开(公告)日：2010-02-04
申请号：US12085873
申请日：2006-11-30
申请人： Marie Denise Parker , Julian Roy Pratt , Yijia Liu , Yang Lu , Martin Woodle , Yuefeng Xie
发明人： Marie Denise Parker , Julian Roy Pratt , Yijia Liu , Yang Lu , Martin Woodle , Yuefeng Xie
IPC分类号： A01N1/02 , C07H21/04
CPC分类号： C12N15/1136 , C12N15/111 , C12N15/113 , C12N15/1138 , C12N2310/11 , C12N2310/14 , C12N2310/53 , C12N2320/31 , C12N2320/32
摘要： This invention describes compositions and methods using siRNA to target various genes expressed in cells of transplanted organs or tissues and/or genes expressed in the host to improve the success of the transplantation.
摘要翻译：本发明描述了使用siRNA靶向在宿主中表达的移植器官或组织和/或基因的细胞中表达的各种基因以提高移植成功的组合物和方法。

8. 发明申请

US20070087047A1 Enhanced circulation effector composition and method 审中-公开
公开(公告)号：US20070087047A1
公开(公告)日：2007-04-19
申请号：US11633129
申请日：2006-12-01
申请人： Samuel Zalipsky , Martin Woodle , Francis Martin , Yechezkel Barenholz , Herve Bercovier
发明人： Samuel Zalipsky , Martin Woodle , Francis Martin , Yechezkel Barenholz , Herve Bercovier
IPC分类号： A61K38/21 , A61K38/20 , A61K9/127
CPC分类号： A61K9/0019 , A61K9/1271 , A61K31/00 , A61K38/12 , A61K38/1774 , A61K47/61 , A61K47/62 , A61K47/6911 , Y10S424/812 , A61K2300/00
摘要： A liposome composition comprising small, surface-bound effector molecules is disclosed. The liposomes have a surface layer of hydrophilic polymer chains, for enhanced circulation time in the bloodstream. The effector molecules are attached to the distal ends of the polymer chains. In one embodiment, the effector is polymyxin B, for treatment of septic shock.

9. 发明授权

US08691781B2 Compositions for treating respiratory viral infections and their use 有权
标题翻译：用于治疗呼吸道病毒感染及其用途的组合物
公开(公告)号：US08691781B2
公开(公告)日：2014-04-08
申请号：US11792179
申请日：2005-11-04
申请人： Qingquan Tang , Patrick Lu , Martin Woodle , Bojian Zheng
发明人： Qingquan Tang , Patrick Lu , Martin Woodle , Bojian Zheng
IPC分类号： A01N43/04 , C12Q1/68 , C12N15/58 , C07H21/02 , C07H21/04
CPC分类号： A61K31/711 , A61K31/7105 , C12N15/1131 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2760/16111 , C12N2760/18511
摘要： The invention provides siRNA compositions that interfere with viral replication in respiratory viral infections, including respiratory syncytial virus and avian influenza A, including the H5N1 strain. The invention further provides uses of the siRNA compositions to inhibit expression of viral genes in respiratory virus-infected cells, and to uses in the treatment of respiratory virus infections in a subject. Generally the invention provides polynucleotide that includes a first nucleotide sequence of 15 to 30 bases that targets the genome of a respiratory syncytial virus or an influenza A virus, a complement thereof, a double stranded polynucleotide or a hairpin polynucleotide. Additionally the invention provides vectors, cells and pharmaceutical compositions containing siRNA sequences.
摘要翻译：本发明提供干扰呼吸道病毒感染（包括呼吸道合胞病毒和禽流感A）包括H5N1毒株在内的病毒复制的siRNA组合物。本发明还提供siRNA组合物用于抑制呼吸道病毒感染细胞中病毒基因表达的用途，以及用于治疗受试者中的呼吸道病毒感染的用途。本发明通常提供多核苷酸，其包含15至30个碱基的第一核苷酸序列，其靶向呼吸道合胞病毒或甲型流感病毒的基因组，其补体，双链多核苷酸或发夹多核苷酸。另外，本发明提供了含有siRNA序列的载体，细胞和药物组合物。

10. 发明申请

US20070203082A1 RNAI Agents For Anti-SARS Coronavirus Therapy 审中-公开
标题翻译：用于抗SARS冠状病毒治疗的RNAI试剂
公开(公告)号：US20070203082A1
公开(公告)日：2007-08-30
申请号：US10554442
申请日：2004-04-26
申请人： Quinn Tang , Patrick Lu , Frank Xie , Yija Liu , Jun Xu , Martin Woodle
发明人： Quinn Tang , Patrick Lu , Frank Xie , Yija Liu , Jun Xu , Martin Woodle
IPC分类号： A61K48/00 , C12Q1/70 , C12Q1/68 , C07H21/02
CPC分类号： C12Q1/701 , C12N15/1131 , C12N2310/14 , C12N2310/53
摘要： The present invention provides compositions and methods that are useful for the treatment of severe acute respiratory syndrome (SARS). More specifically, nucleic acid agents such as siRNA molecules and their analogues that target respiratory infections including SARS coronavirus and their methods of use are described, for clinical treatments of SARS, respiratory viral infections, for prevention and treatment of respiratory infections as needed for bio-defense, for treatment of respiratory diseases, and for discovery of therapeutic targets for respiratory diseases and infections.
摘要翻译：本发明提供可用于治疗严重急性呼吸综合征（SARS）的组合物和方法。更具体地，描述了靶向呼吸道感染（包括SARS冠状病毒及其使用方法）的siRNA分子及其类似物的核酸试剂，用于SARS，呼吸道病毒感染的临床治疗，用于预防和治疗呼吸道感染，防治呼吸道疾病治疗，以及发现呼吸系统疾病和感染的治疗靶点。

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