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1. 发明申请

WO2012135831A1 CELL-PENETRATING ANTI-DNA ANTIBODIES AND USES THEREOF INHIBIT DNA REPAIR 审中-公开
标题翻译：细胞穿透抗DNA抗体及其抑制DNA修复的用途
公开(公告)号：WO2012135831A1
公开(公告)日：2012-10-04
申请号：PCT/US2012/031860
申请日：2012-04-02
申请人： YALE UNIVERSITY , HANSEN, James, E. , GLAZER, Peter, M. , WEISBART, Richard, H. , NISHIMURA, Robert, N. , CHAN, Grace
发明人： HANSEN, James, E. , GLAZER, Peter, M. , WEISBART, Richard, H. , NISHIMURA, Robert, N. , CHAN, Grace
IPC分类号： C07K16/44
CPC分类号： C07K16/18 , A61K31/337 , A61K31/475 , A61K31/513 , A61K31/704 , A61K31/7068 , A61K33/24 , A61K39/39558 , A61K45/06 , A61K2039/505 , C07K16/44 , C07K2317/622 , C07K2317/73 , C07K2317/77 , A61K2300/00
摘要： Antibodies that penetrate cell nuclei and inhibit DNA repair or interfere with DNA metabolism are provided for treatment of cancer (both directly and by sensitizing cancer cells to DNA-damaging treatments) or inhibiting or preventing viral infection, proliferation or metabolism. The method involves treating cells with a composition containing cell-penetrating anti-DNA antibodies or derivatives thereof, alone or in combination with treatment that induces DNA damage such as DNA-damaging chemotherapy or radiation. The impact of the cell-penetrating anti-DNA antibodies or derivatives thereof is potentiated in cancer cells that are deficient in DNA repair, and the cell-penetrating anti-DNA antibodies or derivatives thereof are synthetically lethal to cancer cells with DNA repair deficiencies.
摘要翻译：提供穿透细胞核并抑制DNA修复或干扰DNA代谢的抗体用于治疗癌症（直接和通过致敏癌细胞对DNA损伤性治疗）或抑制或预防病毒感染，增殖或代谢。该方法包括用包含细胞穿透性抗DNA抗体或其衍生物的组合物单独或与诱导DNA损伤如DNA损伤化学疗法或辐射的治疗组合处理细胞。细胞穿透性抗DNA抗体或其衍生物的影响在DNA修复缺陷的癌细胞中被增强，并且细胞穿透性抗DNA抗体或其衍生物对具有DNA修复缺陷的癌细胞是合成致死的。

2. 发明申请

WO2011133802A1 COMPOSITIONS AND METHODS FOR TREATMENT OF LYSOSOMAL STORAGE DISORDERS 审中-公开
标题翻译：用于治疗LYSOSOMAL存储障碍的组合物和方法
公开(公告)号：WO2011133802A1
公开(公告)日：2011-10-27
申请号：PCT/US2011/033475
申请日：2011-04-21
申请人： HELIX THERAPEUTICS, INC. , DEL CAMPO, Jacob , BINDRA, Ranjit, S. , GLAZER, Peter, M.
发明人： DEL CAMPO, Jacob , BINDRA, Ranjit, S. , GLAZER, Peter, M.
IPC分类号： C12N15/113 , C12N15/10 , C12N15/11 , A61K48/00
CPC分类号： A61K48/005 , C12N15/113 , C12N2310/152 , C12N2310/3181 , C12N2310/3519
摘要： Compositions and methods for treating lysosomal storage diseases are disclosed. Lysosomal dysfunction is usually the result of deficiency of a single enzyme necessary for the metabolism of lipids, glycoproteins (sugar containing proteins) or mucopolysaccharides which are fated for breakdown or recycling. The compositions contain triplex-forming molecules which can be used to induce site-specific homologous recombination in mammalian cells when combined with donor DNA molecules, by stimulating cellular DNA synthesis, recombination, and repair mechanisms. The methods are particular useful for correcting point mutations in genes associated with lysosomal storage diseases such as Gaucher' s disease, Fabry disease, and Hurler syndrome. Methods for determining the frequency of target gene repair and assessing the restoration of the enzymatic activity of corrected polypeptides are also disclosed. Ex vivo and in vivo methods of gene correction in patients are also provided.
摘要翻译：公开了用于治疗溶酶体贮积病的组合物和方法。溶酶体功能障碍通常是脂肪，糖蛋白（含糖蛋白）或粘多糖的代谢所必需的单一酶的缺乏的结果，这些蛋白质被命运用于分解或再循环。组合物含有三重形成分子，其可以通过刺激细胞DNA合成，重组和修复机制与哺乳动物细胞中的供体DNA分子结合来诱导位点特异性同源重组。该方法特别适用于校正与溶酶体储存疾病如戈谢氏病，法布里病和赫勒综合征相关的基因中的点突变。还公开了确定靶基因修复频率和评估校正多肽的酶活性恢复的方法。还提供了患者基因修复的离体和体内方法。

3. 发明申请

WO2011053989A2 POLYMERIC MATERIALS LOADED WITH MUTAGENIC AND RECOMBINAGENIC NUCLEIC ACIDS 审中-公开
标题翻译：用MUTAGENIC和重组生物核酸装载的聚合材料
公开(公告)号：WO2011053989A2
公开(公告)日：2011-05-05
申请号：PCT/US2010/055142
申请日：2010-11-02
申请人： YALE UNIVERSITY , SALTZMAN, William, Mark , GLAZER, Peter, M. , CHIN, Joanna , McNEER, Nicole
发明人： SALTZMAN, William, Mark , GLAZER, Peter, M. , CHIN, Joanna , McNEER, Nicole
IPC分类号： A61K48/00
CPC分类号： C12N15/88 , A61K9/0019 , A61K9/1647 , A61K9/19
摘要： Polymeric rnicroparticles are used to deliver recombinagenic or mutagenic nucleic acid molecules such as donor nucleic acid alone, or in combination with triplex forming molecules, to induce a site-specific mutation in the target DNA. Target cells endocytose the particles, releasing the nucleic acid molecules inside of the cell, where they induce mutagenesis or recombination at a target site. The examples demonstrate that triplex forming oligonucleotides, preferably PNAs, preferably in combination with a donor nucleotide molecule, can be encapsulated into polymeric microparticles, which are delivered into cells. Results demonstrate significantly greatly levels of uptake and expression, and less cytotoxicity, as compared to direct transfer of the nucleic acid molecules into the cell by nucleofection.
摘要翻译：聚合物颗粒用于递送重组或诱变核酸分子，例如单独的供体核酸，或与形成三链体的分子组合，以在靶DNA中诱导位点特异性突变。靶细胞吞噬颗粒，释放细胞内的核酸分子，在靶区域诱导诱变或重组。这些实施例表明，三重形成寡核苷酸，优选PNA，优选与供体核苷酸分子组合，可以被包封到聚合物微粒中，其被递送到细胞中。与通过核转染将核酸分子直接转移到细胞中相比，结果显示出显着的摄取和表达水平以及更少的细胞毒性。

4. 发明申请

WO2008086529A3 COMPOSITIONS AND METHODS FOR TARGETED INACTIVATION OF HIV CELL SURFACE RECEPTORS 审中-公开
标题翻译： HIV细胞表面受体靶向灭活的组合物和方法
公开(公告)号：WO2008086529A3
公开(公告)日：2009-02-19
申请号：PCT/US2008050920
申请日：2008-01-11
申请人： UNIV YALE , GLAZER PETER M , BINDRA RANJIT , SCHLEIFMAN ERICA B
发明人： GLAZER PETER M , BINDRA RANJIT , SCHLEIFMAN ERICA B
IPC分类号： A61P31/18 , C12N15/10 , C12N15/11 , C12N15/113
CPC分类号： C12N15/1138 , C12N15/111 , C12N2310/15 , C12N2310/3181 , C12N2310/3519 , C12N2320/30
摘要： Compositions for targeted mutagenesis of cell surface receptors for HIV and methods of their use are provided herein. The compositions include triplex-forming molecules that bind to duplex DNA in a sequence specific manner at target sites to form triple-stranded structures. The triplex-forming molecules can be triplex-forming oligonucleotides (TFOs) or peptide nucleic acids (PNAs). The triplex-forming molecules are useful to induce site-specific homologous recombination in mammalian cells when used in combination with donor oligonucleotides. The triplex-forming molecules target sites within or adjacent to genes that encodes cell surface receptors for human immunodeficiency virus (HIV). This binding stimulates homologous recombination of a donor oligonucleotide to cause mutations in HIV cell surface receptor genes that result in one or more deficiencies in the ability of the encoded receptor to bind to HIV and allow its transport into the cell. Methods for ex vivo and in vivo prophylaxis and therapy of HIV infection using the disclosed compositions are also provided.
摘要翻译：本文提供了用于HIV的细胞表面受体的靶向诱变的组合物及其使用方法。组合物包括在靶位点以序列特异性方式结合双链体DNA的三链体形成分子以形成三链结构。三链体形成分子可以是三重复合形成寡核苷酸（TFO）或肽核酸（PNA）。当与供体寡核苷酸组合使用时，三重形成分子可用于在哺乳动物细胞中诱导位点特异性同源重组。三重形成分子靶向编码人免疫缺陷病毒（HIV）细胞表面受体的基因内或邻近的位点。该结合刺激供体寡核苷酸的同源重组以引起HIV细胞表面受体基因中的突变，其导致编码的受体结合HIV并允许其转运到细胞中的能力的一个或多个缺陷。还提供了使用所公开的组合物进行体外和体内预防和治疗HIV感染的方法。

5. 发明申请

WO2008086529A2 COMPOSITIONS AND METHODS FOR TARGETED INACTIVATION OF HIV CELL SURFACE RECEPTORS 审中-公开
标题翻译： HIV细胞表面受体靶向灭活的组合物和方法
公开(公告)号：WO2008086529A2
公开(公告)日：2008-07-17
申请号：PCT/US2008/050920
申请日：2008-01-11
申请人： YALE UNIVERSITY , GLAZER, Peter, M. , BINDRA, Ranjit , SCHLEIFMAN, Erica, B.
发明人： GLAZER, Peter, M. , BINDRA, Ranjit , SCHLEIFMAN, Erica, B.
IPC分类号： C12N15/11 , C12N15/10 , A61P31/18
CPC分类号： C12N15/1138 , C12N15/111 , C12N2310/15 , C12N2310/3181 , C12N2310/3519 , C12N2320/30
摘要： Compositions for targeted mutagenesis of cell surface receptors for HIV and methods of their use are provided herein. The compositions include triplex-forming molecules that bind to duplex DNA in a sequence specific manner at target sites to form triple-stranded structures. The triplex-forming molecules can be triplex-forming oligonucleotides (TFOs) or peptide nucleic acids (PNAs). The triplex-forming molecules are useful to induce site-specific homologous recombination in mammalian cells when used in combination with donor oligonucleotides. The triplex-forming molecules target sites within or adjacent to genes that encodes cell surface receptors for human immunodeficiency virus (HIV). This binding stimulates homologous recombination of a donor oligonucleotide to cause mutations in HIV cell surface receptor genes that result in one or more deficiencies in the ability of the encoded receptor to bind to HIV and allow its transport into the cell. Methods for ex vivo and in vivo prophylaxis and therapy of HIV infection using the disclosed compositions are also provided.
摘要翻译：本文提供了用于HIV的细胞表面受体的靶向诱变的组合物及其使用方法。组合物包括在靶位点以序列特异性方式结合双链体DNA的三链体形成分子以形成三链结构。三链体形成分子可以是三重复合形成寡核苷酸（TFO）或肽核酸（PNA）。当与供体寡核苷酸组合使用时，三重形成分子可用于在哺乳动物细胞中诱导位点特异性同源重组。三重形成分子靶向编码人免疫缺陷病毒（HIV）细胞表面受体的基因内或邻近的位点。该结合刺激供体寡核苷酸的同源重组以引起HIV细胞表面受体基因中的突变，其导致编码的受体结合HIV并允许其转运到细胞中的能力的一个或多个缺陷。还提供了使用所公开的组合物进行体外和体内预防和治疗HIV感染的方法。

6. 发明申请

WO2011133803A1 COMPOSITIONS AND METHODS FOR TARGETED INACTIVATION OF HIV CELL SURFACE RECEPTORS 审中-公开
标题翻译： HIV细胞表面受体靶向灭活的组合物和方法
公开(公告)号：WO2011133803A1
公开(公告)日：2011-10-27
申请号：PCT/US2011/033477
申请日：2011-04-21
申请人： HELIX THERAPEUTICS, INC. , YALE UNIVERSITY , DEL CAMPO, Jacob , SCHLEIFMAN, Erica, Beth , BINDRA, Ranjit, S. , GLAZER, Peter, M.
发明人： DEL CAMPO, Jacob , SCHLEIFMAN, Erica, Beth , BINDRA, Ranjit, S. , GLAZER, Peter, M.
IPC分类号： C12N15/113 , C12N15/10 , C12N15/90
CPC分类号： C12N15/1138 , C12N15/111 , C12N2310/15 , C12N2310/3181 , C12N2320/33
摘要： Compositions for targeted mutagenesis of cell surface receptors for HIV and methods of their use are provided herein. The compositions include triplex-forming molecules that displace the polypyrimidine strand of target duplex and form a triple-stranded structure and hybrid duplex in a sequence specific manner with the polypurine strand of the target duplex. The triplex-forming molecules include a mixed-sequence "tail" which increases the stringency of binding to the target duplex, improves the frequency of modification at the target site, and reduces the requirement for a polypurine :polypyrimidine stretch. Methods for using the triplex-forming molecules in combination with one or more donor oligonucleotides for targeted modification of sites within or adjacent to genes that encodes cell surface receptors for human immunodeficiency virus (HIV) are also disclosed. Methods for ex vivo and in vivo prophylaxis and therapy of HIV infection using the disclosed compositions are also provided.
摘要翻译：本文提供了用于HIV的细胞表面受体的靶向诱变的组合物及其使用方法。组合物包括取代目标双链体的聚嘧啶链的三链体形成分子，并以序列特异性方式与目标双链体的多肽链形成三链结构和杂合双链体。三重形成分子包括混合序列“尾”，其增加了与目标双链体的结合的严格性，改善了靶位点修饰的频率，并且降低了对于聚亚胺的聚对亚胺的拉伸。还公开了将三链体形成分子与一种或多种供体寡核苷酸组合用于靶向修饰编码人免疫缺陷病毒（HIV）的细胞表面受体的基因内或邻近的基因的方法。还提供了使用所公开的组合物进行体外和体内预防和治疗HIV感染的方法。

7. 发明申请

WO2011053989A3 PROCESS OF TRANSFECTION OF MUTAGENIC AND RECOMBINANT NUCLEIC ACIDS TO CELLS BY POLYMERIC MATERIALS LOADED THEREWITH 审中-公开
标题翻译：通过加载聚合物将MUTAGENIC和重组核酸转移到细胞的过程
公开(公告)号：WO2011053989A3
公开(公告)日：2012-03-01
申请号：PCT/US2010055142
申请日：2010-11-02
申请人： UNIV YALE , SALTZMAN WILLIAM MARK , GLAZER PETER M , CHIN JOANNA , MCNEER NICOLE
发明人： SALTZMAN WILLIAM MARK , GLAZER PETER M , CHIN JOANNA , MCNEER NICOLE
IPC分类号： A61K9/16 , A61K9/00 , A61K9/50 , A61K9/51 , A61K47/00 , C12N15/00
CPC分类号： C12N15/88 , A61K9/0019 , A61K9/1647 , A61K9/19
摘要： Polymeric rnicroparticles are used to deliver recombinagenic or mutagenic nucleic acid molecules such as donor nucleic acid alone, or in combination with triplex forming molecules, to induce a site-specific mutation in the target DNA. Target cells endocytose the particles, releasing the nucleic acid molecules inside of the cell, where they induce mutagenesis or recombination at a target site. The examples demonstrate that triplex forming oligonucleotides, preferably PNAs, preferably in combination with a donor nucleotide molecule, can be encapsulated into polymeric microparticles, which are delivered into cells. Results demonstrate significantly greatly levels of uptake and expression, and less cytotoxicity, as compared to direct transfer of the nucleic acid molecules into the cell by nucleofection.
摘要翻译：聚合物颗粒用于递送重组或诱变核酸分子，例如单独的供体核酸，或与形成三链体的分子组合，以在靶DNA中诱导位点特异性突变。靶细胞吞噬颗粒，释放细胞内的核酸分子，在靶区域诱导诱变或重组。这些实施例表明，三重形成寡核苷酸，优选PNA，优选与供体核苷酸分子组合，可以被包封到聚合物微粒中，其被递送到细胞中。与通过核转染将核酸分子直接转移到细胞中相比，结果显示出显着的摄取和表达水平以及更少的细胞毒性。

8. 发明申请

WO2010123983A1 COMPOSTIONS AND METHODS FOR TARGETED GENE THERAPY 审中-公开
标题翻译：靶向基因治疗的组合物和方法
公开(公告)号：WO2010123983A1
公开(公告)日：2010-10-28
申请号：PCT/US2010/031888
申请日：2010-04-21
申请人： YALE UNIVERSITY , GLAZER, Peter, M. , DEL CAMPO, Jacob
发明人： GLAZER, Peter, M. , DEL CAMPO, Jacob
IPC分类号： C12N15/113 , A61K48/00 , A61K31/7125 , A61P7/06 , A61P43/00
CPC分类号： C12N15/111 , A61K48/00 , C12N15/113 , C12N2310/152 , C12N2310/3181 , C12N2310/3183 , C12N2310/331 , C12N2310/3511 , C12N2310/3513 , C12N2320/30 , C12N2320/34
摘要： Compositions containing molecules that bind to duplex DNA in a sequence-specific manner to form a triple-stranded structure are disclosed. Triplex-forming molecules facilitate strand displacement and triplex formation, referred to as a "clamp," in which one molecule binds to the target strand by Hoogsteen binding and the other molecule binds to the target strand by Watson-Crick binding in a sequence specific manner. A Watson- Crick binding "tail" added to the end of the Watson-Crick binding portion of the clamp increases the stringency of binding to the target duplex, improves the frequency of modification at the target site, and reduces the requirement for a polypurine:polypyrimidine stretch compared to triplex forming oligonucleotides (TFOs) or peptide nucleic acids (PNAs). When administered in combination with a donor oligonucleotide, triplex-forming molecules can enhance recombination of a donor oligonucleotide at the target site. Ex vivo and in vivo methods of gene correction in patients using the disclosed compositions are also disclosed.
摘要翻译：公开了含有以双链体DNA结合的分子的组合物，其以序列特异性方式形成三链结构。三重形成分子促进链置换和三重形成，称为“钳夹”，其中一个分子通过Hoogsteen结合与靶链结合，另一个分子以序列特异性方式通过Watson-Crick结合结合靶链。在夹具的沃森 - 克里克结合部分末端添加的沃森 - 克里克结合“尾巴”增加了与目标双链体的结合的严格性，提高了靶位点修饰的频率，并降低了对于多巴胺的需求：与三重形成寡核苷酸（TFO）或肽核酸（PNA）相比，聚嘧啶拉伸。当与供体寡核苷酸组合施用时，三重形成分子可以增强靶位点处供体寡核苷酸的重组。还公开了使用所公开的组合物的患者中基因校正的离体和体内方法。

9. 发明申请

WO2012135831A8 CELL-PENETRATING ANTI-DNA ANTIBODIES AND USES THEREOF TO INHIBIT DNA REPAIR 审中-公开
标题翻译：细胞穿透抗DNA抗体及其用于抑制DNA修复的用途
公开(公告)号：WO2012135831A8
公开(公告)日：2013-11-21
申请号：PCT/US2012031860
申请日：2012-04-02
申请人： UNIV YALE , HANSEN JAMES E , GLAZER PETER M , WEISBART RICHARD H , NISHIMURA ROBERT N , CHAN GRACE , UNIV CALIFORNIA , US GOV REPRESENTED BY THE DEPT OF VETERANS AFFAIRS
发明人： HANSEN JAMES E , GLAZER PETER M , WEISBART RICHARD H , NISHIMURA ROBERT N , CHAN GRACE
IPC分类号： C07K16/44
CPC分类号： C07K16/18 , A61K31/337 , A61K31/475 , A61K31/513 , A61K31/704 , A61K31/7068 , A61K33/24 , A61K39/39558 , A61K45/06 , A61K2039/505 , C07K16/44 , C07K2317/622 , C07K2317/73 , C07K2317/77 , A61K2300/00
摘要： Antibodies that penetrate cell nuclei and inhibit DNA repair or interfere with DNA metabolism are provided for treatment of cancer (both directly and by sensitizing cancer cells to DNA-damaging treatments) or inhibiting or preventing viral infection, proliferation or metabolism. The method involves treating cells with a composition containing cell-penetrating anti-DNA antibodies or derivatives thereof, alone or in combination with treatment that induces DNA damage such as DNA-damaging chemotherapy or radiation. The impact of the cell-penetrating anti-DNA antibodies or derivatives thereof is potentiated in cancer cells that are deficient in DNA repair, and the cell-penetrating anti-DNA antibodies or derivatives thereof are synthetically lethal to cancer cells with DNA repair deficiencies.
摘要翻译：提供穿透细胞核并抑制DNA修复或干扰DNA代谢的抗体用于治疗癌症（直接和通过致敏癌细胞对DNA损伤性治疗）或抑制或预防病毒感染，增殖或代谢。该方法包括用包含细胞穿透性抗DNA抗体或其衍生物的组合物单独或与诱导DNA损伤如DNA损伤化学疗法或辐射的治疗组合处理细胞。细胞穿透性抗DNA抗体或其衍生物的影响在DNA修复缺陷的癌细胞中被增强，并且细胞穿透性抗DNA抗体或其衍生物对具有DNA修复缺陷的癌细胞是合成致死的。

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