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31. 发明授权

US06300113B1 Isolation and composition of novel glycosidases 失效
标题翻译：新型糖苷酶的分离和组成
公开(公告)号：US06300113B1
公开(公告)日：2001-10-09
申请号：US08560809
申请日：1995-11-21
申请人： David Landry
发明人： David Landry
IPC分类号： C12N924
CPC分类号： C12Y302/01051 , C12N9/2402 , C12Y302/0105 , Y10S435/91
摘要： Substantially pure glycosidases capable for cleaving selected glycosidic bonds have been described including glycosidases isolated from Xanthomonas and recombinant glycosidases. Substrate specificity of isolated enzymes have been identified for GlcNac&bgr;1-X, Gal&agr;1-3R, Gal&agr;1-6R, Gal&bgr;1-3R, Fuc&agr;-2R, Fuc&agr;1-3R, Fuc&agr;1-4R, Man&agr;1-2R, Man&agr;1-3R, Man&agr;1-6R, Man&bgr;1-4R, Xyl&bgr;1-2R, Glc&bgr;1-4R, and Gal&bgr;1-4R providing improved capability for selectively cleaving a glycosidic linkage in a carbohydrate substrate and for forming modified carbohydrates.
摘要翻译：已经描述了能够切割选择的糖苷键的基本上纯的糖苷酶，其包括从黄单胞菌属和重组糖苷酶分离的糖苷酶。已经鉴定了GlcNacbeta1-X，Galalpha1-3R，Galalpha1-6R，Galbeta1-3R，Fucalpha-2R，Fucalpha1-3R，Fucalpha1-4R，Manalpha1-2R，Manalpha1-3R，Manalpha1-6R，Manbeta1的分离的酶的底物特异性 -4R，Xylbeta1-2R，Glcbeta1-4R和Galbeta1-4R，其提供了选择性地切割碳水化合物底物中的糖苷键并形成修饰的碳水化合物的改善的能力。

32. 发明公开

US20230149564A1 AAV-NAGLU VECTORS FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS IIIB 审中-公开
公开(公告)号：US20230149564A1
公开(公告)日：2023-05-18
申请号：US17907025
申请日：2021-03-22
申请人： The University of North Carolina at Chapel Hill
发明人： Haiyan Fu , Tierra Bobo
IPC分类号： A61K48/00 , C12N9/24 , C12N15/86
CPC分类号： A61K48/005 , C12N9/2402 , C12N15/86 , C12Y302/0105
摘要： This invention relates to viral vectors for delivery of α-N-acetylglucosaminidase (NAGLU) to a subject. In some aspects the NAGLU sequence is optimized for expression in human cells. The invention further relates to methods of using the vector to increase secretion of NAGLU from a cell and for treatment and prevention of mucopolysaccharidosis IIIB.

33. 发明申请

US20180119124A1 CRYSTAL STRUCTURE OF STAPHYLOCOCCUS AUREUS AUTOLYSIN E, METHOD OF PRODUCING THE CRYSTAL AND ITS USE IN SCREENING METHODS 审中-公开
公开(公告)号：US20180119124A1
公开(公告)日：2018-05-03
申请号：US15568923
申请日：2015-04-27
申请人： J. STEFAN INSTITUTE
发明人： Marko MIHELIC , Miha RENKO , Dusan TURK
IPC分类号： C12N9/24 , G01N33/573 , G06F19/16
CPC分类号： C12N9/2402 , C07K2299/00 , C12N9/2462 , C12Y302/0105 , G01N33/573 , G01N2333/31 , G01N2333/924 , G16B15/00
摘要： The invention concerns the determination and evaluation of the crystal structure of autolysin E (AtlE) of Staphylococcus aureus (S. aureus), or a crystallizable fragment of AtlE, a method for producing a crystal of AtlE and the respective crystallization kit, and its use in a method for screening an inhibitor of the N-acetylglucosaminidase activity of AtlE, for obtaining atomic spatial relationship data, and for identifying a binding compound of AtlE, e.g. by in silico screening.

34. 发明申请

US20180105879A1 N-ACETYL-alpha-D-GLUCOSAMINIDASE DEFICIENCY COMPOSITIONS AND METHODS 审中-公开
公开(公告)号：US20180105879A1
公开(公告)日：2018-04-19
申请号：US15730382
申请日：2017-10-11
申请人： ALEXION PHARMACEUTICALS, INC.
发明人： Rui-Ru Ji , Andrew Hutchinson , Nina Jain , Christen D. Forbes
IPC分类号： C12Q1/68 , A61K38/47
CPC分类号： C12Q1/6883 , A61K38/47 , C12Q2600/156 , C12Y302/0105
摘要： Compositions and methods relating to potentially pathogenic mutations in the nucleotide sequence of a human NAGLU gene. Some NAGLU gene variants have been discovered to be associated with reduced N-acetyl-α-D-glucosaminidase (NAGLU) activity.

35. 发明申请

US20170216412A1 RECOMBINANT HUMAN NAGLU PROTEIN AND USES THEREOF 审中-公开
公开(公告)号：US20170216412A1
公开(公告)日：2017-08-03
申请号：US15429309
申请日：2017-02-10
申请人： Alexion Pharmaceuticals, Inc.
发明人： ANTHONY QUINN , Markley C. Leavitt , Zhinan Xia
IPC分类号： A61K38/47 , C12N9/24 , A61K9/00
CPC分类号： A61K38/47 , A61K9/0019 , A61K31/7024 , A61K45/06 , C07K7/06 , C07K7/08 , C12N9/2402 , C12N9/2474 , C12Y302/0105 , A61K2300/00
摘要： The present invention provides compositions comprising an isolated mixture of recombinant human NaGlu proteins in which a substantial amount of the NaGlu proteins in the mixture has increased levels of phosphorylated mannose that confer the proteins to be efficiently internalized into human cells. The present invention also provides methods of producing such mixture of NaGlu proteins, vectors used in transgenesis and expression, host cells harboring such vectors, and methods of isolating and purifying the mixture of NaGlu proteins. The invention further provides methods of treating NaGlu associated diseases.

36. 发明授权

US09283181B2 CNS delivery of therapeutic agents 有权
标题翻译： CNS递送治疗剂
公开(公告)号：US09283181B2
公开(公告)日：2016-03-15
申请号：US13168961
申请日：2011-06-25
申请人： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
发明人： Pericles Calias , Jing Pan , Jan Powell , Lawrence Charnas , Thomas McCauley , Teresa Leah Wright , Richard Pfeifer , Zahra Shahrokh
IPC分类号： A61K38/43 , A61K38/46 , A61K38/47 , A61K9/00 , A61K9/19
CPC分类号： A61K38/465 , A61K9/0085 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/47 , C12N9/2402 , C12Y301/06013 , C12Y302/01045 , C12Y302/0105
摘要： The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.
摘要翻译：本发明提供了用于将治疗剂直接递送至中枢神经系统（CNS）的有效且较少侵入性的方法。在一些实施方案中，本发明提供了包括以下步骤的方法：鞘内施用患有或易受溶酶体酶降低的水平或活性的溶酶体贮积病的受试者，包含溶酶体酶的替代酶的组合物。

37. 发明申请

US20160031964A1 Targeted Therapeutic Lysosomal Enzyme Fusion Proteins and Uses Thereof 有权
公开(公告)号：US20160031964A1
公开(公告)日：2016-02-04
申请号：US14883193
申请日：2015-10-14
申请人： BioMarin Pharmaceutical Inc.
发明人： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
IPC分类号： C07K14/65 , C12N9/24
CPC分类号： C07K14/65 , A61K38/00 , A61K38/47 , C07K2319/06 , C12N9/2402 , C12Y302/0105
摘要： The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.

38. 发明授权

US09044473B2 Delivery of therapeutic compounds to the brain and other tissues 有权
公开(公告)号：US09044473B2
公开(公告)日：2015-06-02
申请号：US11630446
申请日：2004-08-30
申请人： Emil D. Kakkis
发明人： Emil D. Kakkis
IPC分类号： A61K38/47 , C12N9/14 , C12Q1/34
CPC分类号： A61K38/465 , A61K9/0085 , A61K38/47 , C12Y301/06008 , C12Y302/0105
摘要： The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human α-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.

39. 发明申请

US20140271598A1 METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE 有权
标题翻译：用于中枢神经系统递送伊曲诺钠硫酸酯的方法和组合物
公开(公告)号：US20140271598A1
公开(公告)日：2014-09-18
申请号：US13974457
申请日：2013-08-23
申请人： Shire Human Genetic Therapies, Inc.
发明人： Gaozhong Zhu , Kris Lowe , Zahra Shahrokh , James Christian , Rick Fahrner , Jing Pan , Teresa Leah Wright , Pericles Calias
IPC分类号： A61K38/46
CPC分类号： A61K38/47 , A61K9/0019 , A61K9/0085 , A61K9/08 , A61K9/19 , A61K35/76 , A61K35/761 , A61K38/46 , A61K38/465 , A61K47/02 , A61K47/26 , C07K14/65 , C12N9/2402 , C12N9/2437 , C12Y301/06008 , C12Y301/06013 , C12Y302/01045 , C12Y302/01046 , C12Y302/0105 , C12Y310/01001
摘要： The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome.
摘要翻译：本发明尤其提供了用于CNS递送溶酶体酶以有效治疗溶酶体贮积病的组合物和方法。在一些实施方案中，本发明包括用于直接CNS鞘内施用的稳定制剂，其包含用于治疗猎人综合征的艾杜糖醛酸-2-硫酸酯酶（I2S）蛋白质，盐和聚山梨醇酯表面活性剂。

40. 发明申请

US20120331578A1 Method for Producing Recombinant Glycoproteins Having Beta1,2-Bound Xylose Residues 审中-公开
标题翻译：生产具有β1,2-结构的木糖残留的重组糖蛋白的方法
公开(公告)号：US20120331578A1
公开(公告)日：2012-12-27
申请号：US13428777
申请日：2012-03-23
申请人： Josef Glössl , Richard Strasser , Jan Mucha , Lukas Mach , Friedrich Altmann , Iain B. Wilson , Herta Steinkellner
发明人： Josef Glössl , Richard Strasser , Jan Mucha , Lukas Mach , Friedrich Altmann , Iain B. Wilson , Herta Steinkellner
IPC分类号： C12P21/00 , C12N5/04 , A01N57/16 , C12N15/82
CPC分类号： C12P21/005 , C12N9/1077 , C12N15/8257 , C12Y204/02038 , C12Y302/01024 , C12Y302/0105 , C12Y302/01052
摘要： A DNA molecule is provided which comprises a sequence according to SEQ ID NO: 8 having an open reading frame from base pair 227 to base pair 1831 or having at least 50% homology to the above-indicated sequence, or hybridizing with the above-indicated sequence under stringent conditions, or comprising a sequence which has degenerated to the above-indicated DNA sequence because of the genetic code, the sequence coding for a plant protein having β1,2-xylosyltransferase activity or being complementary thereto.
摘要翻译：提供了一种DNA分子，其包含根据SEQ ID NO：8的序列，其具有从碱基对227至碱基对1831的开放阅读框，或与上述序列具有至少50％的同源性，或与上述杂交或者包含由于遗传密码而变性为上述DNA序列的序列，编码具有“1,2-木糖基转移酶活性”或与其互补的植物蛋白的序列。

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