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公开(公告)号：US20110262399A1
公开(公告)日：2011-10-27
申请号：US13121532
申请日：2009-09-29
申请人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
发明人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
CPC分类号： C12N9/88 , A61K38/00 , A61K48/0058 , C12N2799/025 , C12Y205/01061
摘要： The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.
摘要翻译： 本发明涉及编码针对哺乳动物细胞中较高表达的人胆色素原脱氨酶的核苷酸序列。 本发明还涉及包含用于基因治疗由胆毒素原脱氨酶（例如急性间歇性卟啉症）缺乏引起的病症的优化的合成编码序列的DNA构建体。 因此，本发明涉及包含编码人胆色素原脱氨酶的核苷酸序列的核酸或核酸构建体，其中至少320个编码人胆色素原脱氨酶的密码子与SEQ ID NO：1中的密码子相同 或其中至少305个编码人胆色素原脱氨酶的密码子与SEQ ID NO：3中的密码子相同。
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公开(公告)号：US08697665B2
公开(公告)日：2014-04-15
申请号：US13121532
申请日：2009-09-29
申请人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
发明人： Antonio Fontanellas Romá , Gloria González Aseguinolaza , Maria Sol Rodriguez Pena , Maria Astrid Pañeda Rodriguez , Jaap Twisk , Jesús Maria Prieto Valtueña , Harald Petry , Sander Jan Hendrik Van Deventer
CPC分类号： C12N9/88 , A61K38/00 , A61K48/0058 , C12N2799/025 , C12Y205/01061
摘要： The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.
摘要翻译： 本发明涉及编码针对哺乳动物细胞中更高表达的人胆色素原脱氨酶的核苷酸序列。 本发明还涉及包含用于基因治疗由胆毒素原脱氨酶（例如急性间歇性卟啉症）缺乏引起的病症的优化的合成编码序列的DNA构建体。 因此，本发明涉及包含编码人胆色素原脱氨酶的核苷酸序列的核酸或核酸构建体，其中至少320个编码人胆色素原脱氨酶的密码子与SEQ ID NO：1中的密码子相同 或其中至少305个编码人胆色素原脱氨酶的密码子与SEQ ID NO：3中的密码子相同。
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3.发明申请METHODS AND COMPOSITIONS FOR THE TREATMENT OF CIRRHOSIS AND LIVER FIBROSIS 审中-公开用于治疗血栓形成和肝纤维化的方法和组合物公开(公告)号：US20120093775A1
公开(公告)日：2012-04-19
申请号：US13260654
申请日：2010-03-26
申请人： Maria Purificaion Fortes Alonso , Jesús Maria Prieto Valtueña , Luciano Matías Sobrevals , Harald Petry , Eric Jacobus Hubertus Timmermans
发明人： Maria Purificaion Fortes Alonso , Jesús Maria Prieto Valtueña , Luciano Matías Sobrevals , Harald Petry , Eric Jacobus Hubertus Timmermans
CPC分类号： A61K35/76 , A61K48/005 , A61K48/0058 , A61K48/0075 , C07K14/65 , C12N7/00 , C12N15/86 , C12N2710/14143 , C12N2750/14143 , C12N2770/22043 , C12N2830/008
摘要： The invention provides a method for the treatment of cirrhosis and liver fibrosis by the use or viral vectors containing the gene encoding IGF-I. The invention discloses both parvoviral vectors and SV40-based vectors as well uses thereof for the treatment of cirrhosis and gene therapy and methods for the preparation of said viral vectors.
摘要翻译： 本发明提供了通过使用含有编码IGF-1的基因的病毒载体来治疗肝硬化和肝纤维化的方法。 本发明公开了小细胞病毒载体和基于SV40的载体以及其用于治疗肝硬化和基因治疗的用途以及用于制备所述病毒载体的方法。
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4.发明申请ONCOSTATIN M AS PROMOTER OF IMMUNOSTIMULATORY ACTIVITY OF HUMAN EPITHELIAL CELLS 审中-公开ONCOSTATIN M作为人上皮细胞免疫活性的促进剂公开(公告)号：US20110195047A1
公开(公告)日：2011-08-11
申请号：US13123031
申请日：2009-10-06
申请人： Rafael Aldabe Arregui , Iranzu González De La Tajada , Maria Esther Larrea Leoz , Jesús Maria Prieto Valtueña , Pablo Sarobe Ugarriza
发明人： Rafael Aldabe Arregui , Iranzu González De La Tajada , Maria Esther Larrea Leoz , Jesús Maria Prieto Valtueña , Pablo Sarobe Ugarriza
CPC分类号： A61K39/39 , A61K38/204 , A61K38/21 , A61K2039/5158 , A61K2039/55522 , A61K2039/57 , A61K2300/00
摘要： The invention relates to the use of oncostatin M (OSM), an OSM receptor (OSMR) agonist, or a combination of OSM or a OSMR agonist with an interferon type I; in the manufacture of a medicament for enhancing the immunostimulatory activity of epithelial cells in a human.
摘要翻译： 本发明涉及制瘤素M（OSM），OSM受体（OSMR）激动剂或OSM或OSMR激动剂与I型干扰素的组合的用途; 在制备用于增强人中上皮细胞的免疫刺激活性的药物中。
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公开(公告)号：US08524860B2
公开(公告)日：2013-09-03
申请号：US12743616
申请日：2008-11-14
申请人： Inés Noelia Casares Lagar , Francisco Borrás Cuesta , Pablo Sarobe Ugarriza , Jesús Maria Prieto Valtueña , Juan José Lasarte Sagastibelza
发明人： Inés Noelia Casares Lagar , Francisco Borrás Cuesta , Pablo Sarobe Ugarriza , Jesús Maria Prieto Valtueña , Juan José Lasarte Sagastibelza
IPC分类号： C07K2/00
CPC分类号： C12N5/0636 , C07K7/08
摘要： The invention relates to peptides of general formula (I), wherein X is absent or X is present and is X14 or X14-X15, wherein X14 and X15, independently from one another, represent an amino acid; their functional variants and fragments, and their pharmaceutically acceptable salts, having the capacity to bind to scurfin and inhibit its biological activity, therefore they regulate or block the activity of regulatory T (Treg) lymphocytes. They are applicable in the treatment of infectious and neoplastic diseases. (I) Arg-Asp-Phe-Gln-Ser-Phe-Arg-Lys-Met-Trp-Pro-Phe- Phe-X
摘要翻译： 本发明涉及通式（I）的肽，其中X不存在或X存在且为X 14或X 14 -X 15，其中X 14和X 15彼此独立地表示氨基酸; 它们的功能变体和片段及其药学上可接受的盐，具有与scurfin结合并抑制其生物活性的能力，因此它们调节或阻断调节性T（Treg）淋巴细胞的活性。 它们适用于感染性和肿瘤性疾病的治疗。 （I）Arg-Asp-Phe-Gln-Ser-Phe-Arg-Lys-Met-Trp-Pro-Phe-Phe-X
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6.发明申请COMPOSTIONS DESIGNED FOR THE INHIBITION AND/OR BLOCKING OF THE EPITHELIAL/MESENCHYMAL TRANSITION 审中-公开用于上传/ MESENCHYMAL过渡的抑制和/或阻塞的组合物公开(公告)号：US20120220546A1
公开(公告)日：2012-08-30
申请号：US13462991
申请日：2012-05-03
申请人： Matías Antonio Avila Zaragoza , Jesús Maria Bañales Asurmendi , Maria Carmen Berasain Lasarte , Fernando José Corrales Izquierdo , MarÍa del Carmen Gil Puig , María Ujue Latasa Sada , Jon Lecanda Cordero , Jesús Maria Prieto Valtueña , Carlos Manuel Rodríguez Ortigosa
发明人： Matías Antonio Avila Zaragoza , Jesús Maria Bañales Asurmendi , Maria Carmen Berasain Lasarte , Fernando José Corrales Izquierdo , MarÍa del Carmen Gil Puig , María Ujue Latasa Sada , Jon Lecanda Cordero , Jesús Maria Prieto Valtueña , Carlos Manuel Rodríguez Ortigosa
IPC分类号： A61K31/7076 , A61P1/16 , A61P35/00 , C07H19/167
CPC分类号： A61K31/7076 , A61K31/00 , A61K45/06 , A61K2300/00
摘要： 5′-methylthioadenosine (MTA) is described as a compound that is susceptible to inhibiting and/or blocking the epithelial-mesenchymal transition (EMT), a process whereby epithelial cells become mesenchymal cells.The periodical intake of MTA [every 24 h for 21 days] significantly improves fibrosis and the markers for hepatic cellular damage in KO-Mdr2 mice with MTA (28 mg/kg). Following the daily oral administration of MTA, both the expression of EMT markers in the total liver and appreciable signs of fibrosis are significantly reduced, indicating the beneficial effect of MTA on the liver affected by the lack of Mdr2. MTA is proposed to be a safe drug, suitable for oral formulation and without secondary effects, to be used in the prevention and/or treatment of diseases associated with EMT, including chronic cholestatic diseases, fibrosis and cholangiocarcinoma. On the other hand, MTA is proposed for application in anti-tumor therapies by inhibiting or blocking the EMT properties of CSC cells, in order to improve the prognosis of tumor development and the malignancy thereof.
摘要翻译： 5'-甲基硫代腺苷（MTA）被描述为易于抑制和/或阻断上皮 - 间质转化（EMT）的化合物，其是上皮细胞成为间充质细胞的过程。 MTA（28天/ 21天）的定期摄入显着改善了MTA（28mg / kg）的KO-Mdr2小鼠的纤维化和肝细胞损伤的标志物。 在每日口服MTA后，EMT标志物在总肝中的表达和纤维化的明显迹象均显着降低，表明MTA对缺乏Mdr2的肝脏有益效果。 MTA被认为是一种安全的药物，适用于口服制剂和无次要作用，用于预防和/或治疗与EMT有关的疾病，包括慢性胆汁淤积性疾病，纤维化和胆管癌。 另一方面，通过抑制或阻断CSC细胞的EMT特性，提出MTA用于抗肿瘤治疗，以改善肿瘤发展的预后及其恶性。
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公开(公告)号：US20110293557A1
公开(公告)日：2011-12-01
申请号：US12997829
申请日：2009-06-12
IPC分类号： A61K38/20 , C07K14/555 , C07H21/04 , C12N15/63 , C12N5/00 , A61P1/16 , A61K38/00 , A61P25/16 , A61P35/00 , A61P17/00 , A61P31/04 , A61P33/00 , A61P19/02 , A61K39/00 , C07K14/775
CPC分类号： A61K38/1709 , A61K45/06 , A61K47/64 , C07K14/47 , C07K14/5434 , C07K14/56 , C07K14/775
摘要： The invention relates to a conjugate that comprises an Apo A molecule or a functionally equivalent variant thereof and a compound of therapeutic interest wherein both components are covalently coupled as well as to the use of said conjugates in therapy for the specific targeting of said compounds to those tissues showing specific binding sites for the ApoA molecule.
摘要翻译： 本发明涉及包含Apo A分子或其功能等同变体的缀合物和治疗感兴趣的化合物，其中两种组分共价偶联，以及所述缀合物在治疗中用于所述化合物对这些化合物的特异性靶向的用途 显示ApoA分子特异性结合位点的组织。
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