公开(公告)号：WO2018045022A1

公开(公告)日：2018-03-08

申请号：PCT/US2017/049359

申请日：2017-08-30

申请人： UNIVERSITY OF SOUTH FLORIDA ,  THE UNITED STATES DEPARTMENT OF VETERANS AFFAIRS

发明人： COOPER, Denise, R. ,  GOULD, Lisa ,  PATEL, Niketa, A. ,  WU, Mack

IPC分类号： A61K35/35 ,  C12N5/00 ,  C12N15/113

CPC分类号： A61K35/35 ,  A61K9/0014 ,  A61P17/02 ,  C12N5/0653 ,  C12N5/0667 ,  C12N15/113 ,  C12N2310/111 ,  C12N2310/20

摘要： Provided herein are exosomal compositions and exsosomal lncRNA compositions and formulations thereof. Also provided herein are methods of treating a wound in a subject in need thereof that can contain the step of administering an exosomal composition and/or exsosomal lncRNA compositions or formulation thereof to a wound in a subject in need thereof.

摘要翻译： 本文提供了外泌体组合物和外泌体lncRNA组合物及其制剂。 本文还提供治疗有需要的受试者的伤口的方法，其可包含将外泌体组合物和/或外泌体组织lncRNA组合物或其制剂给予需要其的受试者的伤口的步骤。

公开(公告)号：WO2017127127A1

公开(公告)日：2017-07-27

申请号：PCT/US2016/024797

申请日：2016-03-29

申请人： SYED, Mubin I.

发明人： SYED, Mubin I.

IPC分类号： A61P3/04 ,  A61P3/06 ,  C07K7/06 ,  C12N15/113

CPC分类号： C12N15/113 ,  A61B34/20 ,  A61B2034/2051 ,  A61B2034/2063 ,  A61K9/14 ,  A61K33/42 ,  A61K38/45 ,  A61M25/01 ,  C12N7/00 ,  C12N15/1136 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/122 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2320/32 ,  C12Y203/01

摘要： Methods for controlling obesity using minimally-invasive procedures including introducing embolic crystal particles that are naturally occurring and mostly non-toxic salts into the arterial capillaries feeding the sections of the stomach where the appetite inducing hormone, ghrelin, is produced to limit the blood flow to the region reducing appetite; introducing a virus vector or antisense oligonucleotide to inhibit the production of ghrelin and reduce the appetite; and introducing a soluble embolic particle with a virus vector or antisense oligonucleotide which will inhibit the flow of blood initially and then dissolve and release the inhibit vector to the region, generating ghrelin to control the appetite.

摘要翻译： 使用微创手术来控制肥胖症的方法包括将天然存在的并且大部分无毒的盐的栓塞晶体颗粒引入动脉毛细血管中，其中将食欲诱导激素ghrelin， 是为了限制血液流向降低食欲的区域而产生的; 引入病毒载体或反义寡核苷酸以抑制生长素释放肽的产生并降低食欲; 并用病毒载体或反义寡核苷酸引入可溶性栓塞颗粒，所述病毒载体或反义寡核苷酸将最初抑制血液的流动，然后溶解并释放抑制载体至该区域，从而生成生长素释放肽以控制食欲。

公开(公告)号：WO2008153733A8

公开(公告)日：2016-07-14

申请号：PCT/US2008006554

申请日：2008-05-22

申请人： NATURE TECHNOLOGY CORP ,  WILLIAMS JAMES A

发明人： WILLIAMS JAMES A

IPC分类号： C12N15/85 ,  A61K39/00

CPC分类号： A61K48/0066 ,  A61K39/12 ,  A61K39/145 ,  A61K48/00 ,  A61K2039/53 ,  A61K2039/55561 ,  C07K14/005 ,  C12N15/111 ,  C12N15/113 ,  C12N15/117 ,  C12N15/85 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/17 ,  C12N2310/532 ,  C12N2320/30 ,  C12N2320/50 ,  C12N2710/10322 ,  C12N2760/16122 ,  C12N2760/16134 ,  C12N2800/106 ,  C12N2800/107 ,  C12N2830/00

摘要： Improved DNA vaccine plasmids are disclosed that contain novel immunostimulatory RNA compositions. The improved plasmids eliminate all extraneous sequences, incorporate a novel antibiotic free short RNA based selectable marker, increase eukaryotic expression using a novel chimeric promoter, improve yield and stability during bacterial production, and improve immunostimulation. These vectors are utilized in immunization to elicit improved immune responses or therapy to induce type 1 interferon production.

摘要翻译： 公开了包含新型免疫刺激RNA组合物的改进的DNA疫苗质粒。 改进的质粒消除所有外来序列，并结合一种新型的基于抗生素的短基于RNA的可选择标记，使用新的嵌合启动子增加真核表达，提高细菌生产过程中的产量和稳定性，并改善免疫刺激。 这些载体用于免疫以引发改善的免疫应答或诱导1型干扰素生产的治疗。

公开(公告)号：WO2016059239A1

公开(公告)日：2016-04-21

申请号：PCT/EP2015/074066

申请日：2015-10-16

申请人： NOGRA PHARMA LIMITED

发明人： MONTELEONE, Giovanni

IPC分类号： C12N15/113 ,  A61K31/7088 ,  C12Q1/68

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2320/34 ,  C12N2320/35 ,  C12Q1/6883 ,  C12Q2600/156

摘要： The present invention relates to treatment of inflammatory bowel disease (e.g., Crohn's disease and ulcerative colitis) using antisense nucleotides that are directed against polymorphic forms (e.g., those containing single nucleotide polymorphisms) of the SMAD7 mRNA. The invention thus relates to treatment methods for subjects having polymorphic forms of SMAD7 and antisense oligonucleotides that specifically target SMAD7 mRNA transcripts containing polymorphisms.

摘要翻译： 本发明涉及使用针对SMAD7 mRNA的多态性（例如含有单核苷酸多态性的那些）的反义核苷酸治疗炎性肠病（例如克罗恩病和溃疡性结肠炎）。 因此本发明涉及具有特异性形式的SMAD7的受试者和特异性靶向含有多态性的SMAD7 mRNA转录物的反义寡核苷酸的治疗方法。

公开(公告)号：WO2016022526A1

公开(公告)日：2016-02-11

申请号：PCT/US2015/043548

申请日：2015-08-04

申请人： THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

发明人： DMOCHOWSKI, Ivan J. ,  GRIEPENBURG, Julianne C. ,  RAPP, Teresa L.

IPC分类号： B01J31/18

CPC分类号： C07F15/0053 ,  B01J31/1815 ,  B01J35/004 ,  B01J2531/821 ,  C07H23/00 ,  C12N13/00 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/32 ,  C12N2310/3233 ,  C12N2310/351 ,  C12N2310/3517 ,  C12N2310/531 ,  C12Q1/6818 ,  C12Q1/6823 ,  C12Q1/6825

摘要： The present invention provides ruthenium-based photolinker compounds, caged molecules comprising the ruthenium-based photolinker compounds, and methods of use. In certain aspects, the compositions disclosed herein comprise an active domain conjugated to a ruthenium-based photolinker, such that irradiation of the photolinker exposes the active domain. The present invention includes a composition comprising at least one ruthenium-based photolinker compound of formula (II), wherein in formula (II): L1, L2, L3, and L4 are each independently a ligand; and X1 and X2 are each independently a photolabile ligand having a reactive moiety.

摘要翻译： 本发明提供了钌基光接枝剂化合物，包含钌基光接枝剂化合物的笼状分子和使用方法。 在某些方面，本文公开的组合物包含与钌基光引发剂缀合的活性结构域，使得光引发剂的照射暴露于活性域。 本发明包括包含至少一种式（II）的钌基光接枝化合物的组合物，其中在式（II）中：L1，L2，L3和L4各自独立地为配体; X1和X2各自独立地为具有反应性部分的光不稳定配体。

公开(公告)号：WO2015187989A1

公开(公告)日：2015-12-10

申请号：PCT/US2015/034264

申请日：2015-06-04

申请人： ISIS PHARMACEUTICALS, INC. ,  ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE

发明人： RIGO, Frank ,  HASTINGS, Michelle, L.

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/1138 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/33

摘要： The present invention provides compounds comprising oligonucleotides complementary to an LRP8 transcript. Certain such compounds are useful for hybridizing to an LRP8 transcript, including but not limited, to an LRP8 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRP8 transcript. In certain embodiments, such hybridization results in an increase in inclusion of exon 19 in the LRP8 mRNA transcript. In certain embodiments, such compounds are used to treat Alzheimer's Disease.

摘要翻译： 本发明提供了包含与LRP8转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与LRP8转录物（包括但不限于）细胞中的LRP8转录物杂交。 在某些实施方案中，这种杂交导致LRP8转录物剪接的调节。 在某些实施方案中，这种杂交导致外显子19在LRP8 mRNA转录物中的包含增加。 在某些实施方案中，这些化合物用于治疗阿尔茨海默病。

公开(公告)号：WO2015162161A1

公开(公告)日：2015-10-29

申请号：PCT/EP2015/058684

申请日：2015-04-22

申请人： MEDIZINISCHE HOCHSCHULE HANNOVER

发明人： THUM, Thomas ,  REGALLA, Kumarswamy ,  VIERECK, Janika

IPC分类号： C12N15/113 ,  A61K31/7088

CPC分类号： C12N15/113 ,  A61K48/0058 ,  A61P9/00 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/113 ,  C12N2310/14 ,  C12N2320/32 ,  C12Q1/6883 ,  C12Q2600/158 ,  C12Q2600/178

摘要： The present invention relates to a pharmaceutical composition comprising (i) a compound promoting the expression and/or the activity of one or more long non-coding RNAs (lncRNAs) selected from SEQ ID NOs 12, 8 to 11 and 13; and/or (ii) a compound inhibiting the expression and/or the activity of one or more lncRNAs selected from SEQ ID NOs 1 to 7, 27 and 28. The present invention also relates to a compound (i) promoting the expression and/or the activity of one or more lncRNAs selected from SEQ ID NOs 12, 8 to 11 and 13; and/or (ii) inhibiting the expression and/or the activity of one or more lncRNAs selected from SEQ ID NOs 1 to 7, 27 and 28 for use in treating or preventing cardiac hypertrophy.

摘要翻译： 本发明涉及药物组合物，其包含（i）促进选自SEQ ID NO 12,8至11和13的一种或多种长非编码RNA（lncRNA）的表达和/或活性的化合物; 和/或（ii）抑制一种或多种选自SEQ ID NO：1-7,27和28的lncRNA的表达和/或活性的化合物。本发明还涉及促进表达和/ 或选自SEQ ID NO：12,8至11和13的一种或多种lncRNA的活性; 和/或（ii）抑制一种或多种选自SEQ ID NO：1，7，27和28的lncRNA的表达和/或活性，用于治疗或预防心脏肥大。

公开(公告)号：WO2015085113A1

公开(公告)日：2015-06-11

申请号：PCT/US2014/068654

申请日：2014-12-04

申请人： RXI PHARMACEUTICALS CORPORATION

发明人： CAUWENBERGH, Gerard ,  PAVCO, Pamela, A. ,  LIBERTINE, Lyn ,  BULOCK, Karen, G. ,  CARDIA, James

IPC分类号： A61P17/02

CPC分类号： C12N15/1136 ,  A61K9/0014 ,  A61K9/0021 ,  A61K9/0048 ,  A61K31/7088 ,  A61K31/7125 ,  A61K31/713 ,  A61K47/60 ,  C12N15/1137 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/35 ,  A61K2300/00

摘要： The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal and fibrotic applications. Aspects of the invention provide nucleic acid molecules for the prophylactic treatment of wounding to reduce scarring. Herein, it is demonstrated that a specific nucleic acid molecule, RXI-109 (targeting connective tissue growth factor (CTGF)), given prophylactically, reduces scarring during wound healing.

摘要翻译： 本发明涉及具有改善的组织和细胞摄取特性的RNAi构建体以及在真皮和纤维化应用中使用这些化合物的方法。 本发明的方面提供用于预防性治疗伤口以减少瘢痕形成的核酸分子。 在本文中，证明特定核酸分子RXI-109（靶向结缔组织生长因子（CTGF））预防性地减少伤口愈合过程中的瘢痕形成。

公开(公告)号：WO2014193822A1

公开(公告)日：2014-12-04

申请号：PCT/US2014/039549

申请日：2014-05-27

申请人： THE REGENTS OF THE UNIVERSITY OF CALIFORNIA

发明人： MILLER, Yury ,  FANG, Longhou

IPC分类号： A61K38/00 ,  A61P3/06 ,  A61P9/00 ,  C07K14/515

CPC分类号： C07K14/47 ,  A61K38/00 ,  A61K38/1761 ,  C07K16/18 ,  C07K2317/76 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/3233

摘要： The invention provides pharmaceutical compounds and formulations comprising nucleic acids and polypeptides for regulating (including upregulating or inhibiting) the expression of ApoA-l Binding Protein (APOAIBP, AIBP, or AI-BP), and methods for making and using them. In alternative embodiments, APOAIBP-inhibiting pharmaceutical compositions and formulations of the invention are administered to an individual in need thereof in an amount sufficient to stimulate tissue revascularization, e.g., supporting or stimulating revascularization of heart tissue, e.g., after a cardiac ischemia. In alternative embodiments, pharmaceutical compositions and formulations of the invention that comprise APOAIBP nucleic acids and polypeptides or result in an increase in expression or activity of APOAIBP nucleic acids and polypeptides are administered to an individual in need thereof in an amount sufficient to treat, prevent, reverse and/or ameliorate a dyslipidemia, e.g., to treat, prevent, reverse and/or ameliorate conditions responsive to increasing cholesterol efflux from cells, including cardiovascular disease and atherosclerosis.

摘要翻译： 本发明提供包含用于调节（包括上调或抑制）ApoA-1结合蛋白（APOAIBP，AIBP或AI-BP）的表达的核酸和多肽的药物化合物和制剂及其制备和使用方法。 在替代实施方案中，本发明的APOAIBP抑制药物组合物和制剂以足以刺激组织血运重建的量给予有此需要的个体，例如支持或刺激心脏组织的血运重建，例如在心脏缺血后。 在替代实施方案中，包含APOAIBP核酸和多肽或导致APOAIBP核酸和多肽的表达或活性增加的本发明的药物组合物和制剂以足以治疗，预防， 逆转和/或改善血脂异常，例如，治疗，预防，逆转和/或改善对增加胆固醇从细胞外溢（包括心血管疾病和动脉粥样硬化）的反应。

公开(公告)号：WO2014144423A3

公开(公告)日：2014-11-13

申请号：PCT/US2014028830

申请日：2014-03-14

申请人： TECHULON INC

发明人： MALONE BRETT ,  BRYSON JOSHUA

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  A61K9/0014 ,  A61K31/7088 ,  A61K47/64 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/3181 ,  C12N2310/3513 ,  C12N2320/30

摘要： Disclosed are antisense molecules and compositions for the treatment of Staphylococcus aureus infection. The antisense molecules and compositions comprise nucleic acid molecules, such as RNA, DNA, or nucleic acid molecules with modified backbones, such as PNA. The antisense molecules and compositions inhibit gene expression in Staphylococcus aureus; are optionally conjugated to cell penetration molecules such as peptides; and are optionally administered in the form of a nanoparticle composition.

摘要翻译： 公开了用于治疗金黄色葡萄球菌感染的反义分子和组合物。 反义分子和组合物包含核酸分子，例如RNA，DNA或具有修饰的骨架的核酸分子，例如PNA。 反义分子和组合物抑制金黄色葡萄球菌中的基因表达; 任选地缀合到细胞穿透分子如肽; 并且任选地以纳米颗粒组合物的形式施用。