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1. 发明授权

US06537777B1 Human porphobilinogen deaminase sequences 有权
标题翻译：人胆色素原脱氨酶序列
公开(公告)号：US06537777B1
公开(公告)日：2003-03-25
申请号：US09358856
申请日：1999-07-22
申请人： Par Gellerfors , Jens Fogh
发明人： Par Gellerfors , Jens Fogh
IPC分类号： C12P2106
CPC分类号： C12N9/88 , C12Y205/01061
摘要： A method for treatment or prophylaxis of disease caused by deficiency, in a subject, of an enzyme belonging to the heme biosynthetic pathway, the method comprising administering, to the subject, an effective amount of a catalyst which is an enzyme or an enzymatically equivalent part or analogue thereof. The disease is selected from the group consisting of acute intermittent porphyria (AIP), ALA deficiency porphyria (ADP), Porphyria cutanea tarda (PCT), Hereditary coproporphyria (HCP), Harderoporphyria (HDP), Variegata prophyria (VP), Congenital erthropoetic porphyria (CEP), Erythropoietic protoporphyria (EPP), and Hepatoerythropoietic porphyria (HEP). The catalyst is one or more enzymes selected from the group consisting of delta-aminolevulininic acid synthetase, delta-aminolevulinic acid dehydratase (ALAD), porphobilinogen deaminase (PBGD), uroporphyrinogen III cosythetase, uroporphyrinogen decarboxylase, coproporphyrinogen oxidase, protoporphyrinogen oxidase, and ferrochelatase, or an enzymatically equivalent part or analogue thereof. In addition the invention relates to the use of PBGD, to human recombinant PBGD and to a method of gene therapy. The invention also relates to an expression plasmid pExp1-M2-BB (Seq. ID No. 1) and to use of a DNA fragment, the EcoRI-Hind III linear fragment (seq. ID No. 2), used for transformation in the hemC disruption strategy for production of rhPBGD expressed in E. coli.
摘要翻译：一种用于治疗或预防受试者由属于血红素生物合成途径的酶引起的疾病的方法，所述方法包括向所述受试者施用有效量的作为酶或酶等价部分的催化剂或其类似物。该疾病选自急性间歇性卟啉症（AIP），ALA缺乏性卟啉症（ADP），卟啉症绦虫（PCT），遗传性共挫折症（HCP），硬毛霉素（HDP），瓦氏病卟啉症（VP），先天性埃及卟啉症（CEP），红细胞生成原始疟原虫（EPP）和肝造影卟啉症（HEP）。催化剂是一种或多种选自δ-氨基乙酰丙酸合成酶，δ-氨基乙酰丙酸脱水酶（ALAD），胆色素原脱氨酶（PBGD），尿卟啉原III舒适酶，尿卟啉原脱羧酶，cop卟啉原氧化酶，原卟啉原氧化酶和铁螯合酶的一种或多种酶，或其酶等同部分或类似物。此外，本发明涉及PBGD，人重组PBGD和基因治疗方法的用途。本发明还涉及表达质粒pExp1-M2-BB（Seq.ID No.1），并且使用DNA片段，用于转化的EcoRI-Hind III线性片段（seq.ID No.2）用于生产在大肠杆菌中表达的rhPBGD的hemC破坏策略。

2. 发明申请

US20070099185A1 Methods for the identification of agents that modulate the structure and processing of beta-amyloid precursor protein 审中-公开
标题翻译：用于鉴定调节β-淀粉样蛋白前体蛋白的结构和加工的试剂的方法
公开(公告)号：US20070099185A1
公开(公告)日：2007-05-03
申请号：US10533844
申请日：2003-11-04
申请人： Frederick Hagen , Lars Lannfelt , Par Gellerfors
发明人： Frederick Hagen , Lars Lannfelt , Par Gellerfors
IPC分类号： C40B30/06 , C40B40/08 , C40B40/10
CPC分类号： G01N33/5008 , G01N33/6896 , G01N2333/4709 , G01N2800/2821
摘要： The present invention provides methods for the screening and identification of agents from a large library of molecular structures that can alter the cleavage of amyloid precursor protein (AP). Agents identified by the methods of the present invention that modify the cleavage of APP can be used in the treatment and prevention of Alzheimer's disease. The methods select for and identify effector agents that bind to APP causing a structural change in the structure of APP in such a way that the efficiency of the cleavage of a secretase is modulated. Further, the methods are carried out in an in vivo system that provides for physiological conditions similar or identical to conditions for APP processing. Agents can be selected for their ability to cause a decrease in the amount of B-secretase or β-secretase cleavage of APP, or for an increase in a-secretase cleavage of APP. The agents can be, particularly peptide agents, can be converted into a peptidominetic, an isosteric replacement compound, a D-amino acid analog, or non-peptidyl compound for treating Alzheimer's disease or any other amyloid related or prion related disease. The agents or derivatives thereof can be formulated for intravenous, parenteral, topical, sustained release, intranasal, or inhalation use.
摘要翻译：本发明提供了用于筛选和鉴定可以改变淀粉样蛋白前体蛋白（AP）的切割的大分子结构文库的试剂的方法。通过修饰APP切割的本发明方法鉴定的药剂可用于治疗和预防阿尔茨海默氏病。所述方法选择并鉴定结合APP的效应物，其导致APP结构的结构变化，使得调节分泌酶切割的效率。此外，该方法在体内系统中进行，其提供与APP处理的条件相似或相同的生理条件。可以选择代谢物以降低APP的B-分泌酶或β-分泌酶裂解的量，或者增加APP的分泌酶裂解。药剂可以是特别是肽剂，可以转化为用于治疗阿尔茨海默氏病或任何其他与淀粉样蛋白相关或朊病毒相关疾病的肽基质，异构体替代化合物，D-氨基酸类似物或非肽基化合物。药剂或其衍生物可以配制用于静脉内，肠胃外，局部，持续释放，鼻内或吸入使用。

3. 发明授权

US5273966A O-glycosylated IGF-1 失效
标题翻译： O-糖基化的IGF-1
公开(公告)号：US5273966A
公开(公告)日：1993-12-28
申请号：US654611
申请日：1991-04-22
申请人： Anna Sknottner-Lundin , Linda Fryklund , Par Gellerfors
发明人： Anna Sknottner-Lundin , Linda Fryklund , Par Gellerfors
IPC分类号： A61K38/28 , A61K38/00 , A61K38/27 , A61K38/30 , A61P3/08 , A61P3/10 , C07K1/113 , C07K14/00 , C07K14/575 , C07K14/65 , C12N15/09 , C12N15/16 , C12N15/81 , C12P21/02 , C12R1/865 , A61K37/36 , C07K7/40 , C12P21/00
CPC分类号： C12N15/81 , A61K38/30 , C07K14/65
摘要： The present invention provides an O-Glycosylated Insulin-like Growth Factor 1 (IGF-1) analog of Insulin-like Growth Factor 1, a seventy amino acids single polypeptide chain which displays relatively high homology with proinsulin, essentially free from unglycosylated IGF-1.

4. 发明申请

US20090258009A1 PROTOFIBRIL SELECTIVE ANTIBODIES AND THE USE THEREOF 有权
标题翻译：抗原选择性抗体及其用途
公开(公告)号：US20090258009A1
公开(公告)日：2009-10-15
申请号：US12294207
申请日：2007-03-23
申请人： Par Gellerfors , Lars Lannfelt , Dag Sehlin , Frida Ekholm Pettersson , Hillevi Englund
发明人： Par Gellerfors , Lars Lannfelt , Dag Sehlin , Frida Ekholm Pettersson , Hillevi Englund
IPC分类号： A61K39/395 , C07K16/18 , A61P25/28 , A61P25/18 , A61P25/00 , G01N33/566
CPC分类号： G01N33/6896 , A61K2039/505 , C07K16/18 , C07K16/465 , C07K2317/24 , C07K2317/565 , G01N2333/4709 , G01N2800/2814 , G01N2800/387
摘要： The present invention pertains to the prevention, treatment and diagnosis of neurodegenerative diseases, in particular Alzheimer's disease, and other similar disease. More specifically to high affinity antibodies selective for amyloid beta protein (Aβ) in its protofibril conformation and of IgG class and IgG1 or IgG4 subclass or combinations thereof or mutations thereof, retaining high Fc receptor binding and low C1(CIq) binding, effective in clearance of Aβ protofibrils and with reduce risk of inflammation.
摘要翻译：本发明涉及神经变性疾病，特别是阿尔茨海默氏病和其他类似疾病的预防，治疗和诊断。更具体地，涉及其原始原纤维构象中的淀粉样蛋白β蛋白（Abeta）和IgG类和IgG1或IgG4亚类或其组合或其突变选择性的高亲和力抗体，保留高Fc受体结合和低C1（C1q）结合，有效清除的Abeta原纤维，并减少炎症的风险。

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