JP6329483B2 - 組換えヒトｎａｇｌｕタンパク質およびその利用 - Google Patents
組換えヒトｎａｇｌｕタンパク質およびその利用 Download PDFInfo
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- JP6329483B2 JP6329483B2 JP2014535865A JP2014535865A JP6329483B2 JP 6329483 B2 JP6329483 B2 JP 6329483B2 JP 2014535865 A JP2014535865 A JP 2014535865A JP 2014535865 A JP2014535865 A JP 2014535865A JP 6329483 B2 JP6329483 B2 JP 6329483B2
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- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
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| PCT/US2012/059708 WO2013055888A2 (en) | 2011-10-12 | 2012-10-11 | Recombinant human naglu protein and uses thereof |
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| US8759297B2 (en) | 2006-08-18 | 2014-06-24 | Armagen Technologies, Inc. | Genetically encoded multifunctional compositions bidirectionally transported between peripheral blood and the cns |
| AU2008282496B2 (en) | 2007-07-27 | 2013-04-04 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-iduronidase activity in the CNS |
| EP2485761B1 (en) | 2009-10-09 | 2019-02-27 | Armagen, Inc. | Methods and compositions for increasing iduronate 2-sulfatase activity in the cns |
| KR20150038636A (ko) | 2010-09-09 | 2015-04-08 | 시나게바 바이오파르마, 코포레이션 | 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도 |
| WO2012112681A1 (en) | 2011-02-15 | 2012-08-23 | Shire Human Genetic Therapies, Inc. | Methods for treating lysosomal acid lipase deficiency |
| SG10201510106PA (en) * | 2011-10-12 | 2016-01-28 | Synageva Biopharma Corp | Recombinant human naglu protein and uses thereof |
| AU2012346448B2 (en) | 2011-12-02 | 2017-09-14 | Armagen, Inc. | Methods and compositions for increasing arylsulfatase A activity in the CNS |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| TWI793159B (zh) * | 2013-10-23 | 2023-02-21 | 美商健臻公司 | 重組醣蛋白及其用途 |
| CA2947468C (en) * | 2014-05-14 | 2022-10-25 | Laboratorios Del Dr. Esteve S.A. | Adenoassociated virus vectors for the treatment of lysosomal storage disorders |
| US20170267986A1 (en) * | 2014-06-25 | 2017-09-21 | Shire Human Genetic Therapies, Inc. | Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase |
| EP3185889B1 (en) | 2014-08-11 | 2020-07-15 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
| EP3200816A1 (en) * | 2014-09-29 | 2017-08-09 | Alexion Pharmaceuticals, Inc. | Methods of treating mucopolysaccharidosis iiib (mpsiiib) |
| CN104524662B (zh) * | 2014-12-31 | 2017-02-22 | 迈得医疗工业设备股份有限公司 | 一种医用针座组装的上料机构 |
| US10538589B2 (en) * | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
| US20190083583A1 (en) * | 2016-01-29 | 2019-03-21 | Alexion Pharmaceuticals, Inc. | Enzyme replacement therapy in mucopolysaccharidosis iiib patients |
| SG10201912761UA (en) | 2016-04-15 | 2020-02-27 | The Trustees Of The Univ Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| US10633705B2 (en) | 2016-10-14 | 2020-04-28 | Alexion Pharmaceuticals, Inc. | N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| CR20200129A (es) | 2017-10-02 | 2020-08-22 | Denali Therapeutics Inc | Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas |
| US12605465B2 (en) | 2020-03-23 | 2026-04-21 | The University Of North Carolina At Chapel Hill | AAV-NAGLU vectors for treatment of mucopolysaccharidosis IIIB |
| CN111718947B (zh) * | 2020-06-18 | 2022-08-23 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途 |
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| US6825396B2 (en) | 1996-06-12 | 2004-11-30 | Board Of Trustees Operating Michigan State University | Methods for tissue specific synthesis of protein in eggs of transgenic hens |
| US5897998A (en) | 1997-08-04 | 1999-04-27 | The University Of Georgia Research Foundation, Inc. | Method for manipulating avian eggs |
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| US7511120B2 (en) | 1997-10-16 | 2009-03-31 | Synageva Biopharma Corp. | Glycosylated G-CSF obtained from a transgenic chicken |
| JP2001520009A (ja) | 1997-10-16 | 2001-10-30 | ユニバーシティ・オブ・ジョージア・リサーチ・ファウンデイション・インコーポレイテッド | 鳥の導入遺伝子についてのマグナム特異的プロモーターを含むベクター |
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
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| US7294507B2 (en) | 2001-11-30 | 2007-11-13 | Avigenics, Inc. | Ovomucoid promoters and methods of use |
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| ES2371913T3 (es) | 2003-01-22 | 2012-01-11 | Duke University | Constructos mejorados para expresar polipéptidos lisosomales. |
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| US20050090001A1 (en) | 2003-10-07 | 2005-04-28 | Parker Stephen H. | Cell lines and methods for producing proteins |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
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| CA2641070C (en) * | 2006-02-06 | 2017-01-03 | Ramot At Tel-Aviv University Ltd. | Enzyme replacement therapy for treating lysosomal storage diseases |
| CN101410408A (zh) * | 2006-04-04 | 2009-04-15 | 希尔制药爱尔兰有限责任公司 | 用于浓缩多肽的方法 |
| WO2008091311A1 (en) | 2007-01-26 | 2008-07-31 | Synageva Biopharma Corp | Transgene expression in avians |
| CN101932231A (zh) * | 2008-01-07 | 2010-12-29 | 辛那杰瓦生物制药股份有限公司 | 禽类的糖基化 |
| WO2009131698A2 (en) * | 2008-04-23 | 2009-10-29 | Iowa State University Research Foundation, Inc. | PHOSPHORYLATED RECOMBINANT N-ACETYL-alpha-D- GLUCOSAMINIDASE (NaGlu) AND USES THEREOF |
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| LT2588130T (lt) * | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| AU2011270672B2 (en) * | 2010-06-25 | 2017-01-12 | Shire Human Genetic Therapies, Inc. | Treatment of Sanfilippo Syndrome type B |
| US8775146B2 (en) * | 2010-07-22 | 2014-07-08 | Shire Human Genetic Therapies, Inc. | Crystal structure of human alpha-N-acetylglucosaminidase |
| KR20150038636A (ko) | 2010-09-09 | 2015-04-08 | 시나게바 바이오파르마, 코포레이션 | 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도 |
| SG10201510106PA (en) * | 2011-10-12 | 2016-01-28 | Synageva Biopharma Corp | Recombinant human naglu protein and uses thereof |
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| BR112014008873A2 (pt) | 2020-09-15 |
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| EP2766477A4 (en) | 2015-07-15 |
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| WO2013055888A2 (en) | 2013-04-18 |
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| JP2014534962A (ja) | 2014-12-25 |
| NZ623690A (en) | 2016-08-26 |
| EP2766477B1 (en) | 2018-09-26 |
| CN103958676A (zh) | 2014-07-30 |
| US20140255383A1 (en) | 2014-09-11 |
| SG10201510106PA (en) | 2016-01-28 |
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