CN1420924B - 重组α-L-艾杜糖苷酶,其生产和纯化的方法以及治疗其缺陷导致的疾病的方法 - Google Patents

重组α-L-艾杜糖苷酶,其生产和纯化的方法以及治疗其缺陷导致的疾病的方法 Download PDF

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CN1420924B
CN1420924B CN008181705A CN00818170A CN1420924B CN 1420924 B CN1420924 B CN 1420924B CN 008181705 A CN008181705 A CN 008181705A CN 00818170 A CN00818170 A CN 00818170A CN 1420924 B CN1420924 B CN 1420924B
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iduronidase
enzyme
leu
recombinant alpha
ala
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CN1420924A (zh
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J·M·亨德斯特兰德
秦民民
陈炜鹏
陈临
P·A·菲茨帕特里克
D·J·文特
G·N·泽切尔勒
C·M·斯塔尔
E·D·卡基斯
B·塔纳马奇
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HARBOR-UCLA
Biomarin Pharmaceutical Inc
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
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CN008181705A 1999-11-12 2000-11-09 重组α-L-艾杜糖苷酶,其生产和纯化的方法以及治疗其缺陷导致的疾病的方法 Expired - Lifetime CN1420924B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/439,923 US6426208B1 (en) 1999-11-12 1999-11-12 Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US09/439,923 1999-11-12
PCT/US2000/031293 WO2002004616A1 (en) 1999-11-12 2000-11-09 RECOMBINANT α-L-IDURONIDASE, METHODS FOR PRODUCING AND PURIFYING THE SAME AND METHODS FOR TREATING DISEASES CAUSED BY DEFICIENCIES THEREOF

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CNA2008101440588A Division CN101407796A (zh) 1999-11-12 2000-11-09 重组α-L-艾杜糖苷酶,其生产和纯化的方法以及治疗其缺陷导致的疾病的方法

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CN1420924A CN1420924A (zh) 2003-05-28
CN1420924B true CN1420924B (zh) 2010-05-05

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CN008181705A Expired - Lifetime CN1420924B (zh) 1999-11-12 2000-11-09 重组α-L-艾杜糖苷酶,其生产和纯化的方法以及治疗其缺陷导致的疾病的方法
CNA2008101440588A Pending CN101407796A (zh) 1999-11-12 2000-11-09 重组α-L-艾杜糖苷酶,其生产和纯化的方法以及治疗其缺陷导致的疾病的方法

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US (3) US6426208B1 (OSRAM)
EP (2) EP1332206B1 (OSRAM)
JP (3) JP2004502456A (OSRAM)
CN (2) CN1420924B (OSRAM)
AT (2) ATE342964T1 (OSRAM)
AU (2) AU773663B2 (OSRAM)
BR (1) BRPI0015533B8 (OSRAM)
CA (1) CA2391098C (OSRAM)
DE (2) DE60031456T2 (OSRAM)
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MX (1) MXPA02004742A (OSRAM)
WO (1) WO2002004616A1 (OSRAM)
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EP3676385A1 (en) 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ii型を治療するための遺伝子療法
CN107699590A (zh) * 2017-10-13 2018-02-16 成都中医药大学 一种制备重组人α‑L‑艾杜糖醛酸酶的方法
US20220023434A1 (en) 2018-12-19 2022-01-27 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional Molecules for Lysosomal Targeting and Related Compositions and Methods
CN110577559B (zh) * 2019-08-19 2023-02-17 江西科技师范大学 α-L-艾杜糖醛酸酶测定用荧光糖苷酶底物的合成方法
JP7791517B2 (ja) * 2021-01-04 2025-12-24 国立大学法人徳島大学 ライソゾームを標的とした新規ddsの開発
WO2022145449A1 (ja) * 2021-01-04 2022-07-07 国立大学法人徳島大学 ライソゾームを標的とした新規ddsの開発
CN115109790A (zh) * 2021-03-23 2022-09-27 北京据德医药科技有限公司 一种重组a-L-艾杜糖醛酸普酶及其制备方法
JP2024524614A (ja) 2021-07-14 2024-07-05 ライシア セラピューティクス, インコーポレイテッド Asgpr細胞表面受容体結合化合物及びコンジュゲート
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AU2004208651A1 (en) 2004-09-30
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AU8463501A (en) 2002-01-21
IL149568A (en) 2010-04-29
US20030013179A1 (en) 2003-01-16
ZA200203619B (en) 2003-07-30
ATE430193T1 (de) 2009-05-15
US7041487B2 (en) 2006-05-09
BRPI0015533B1 (pt) 2020-12-08
HK1057059A1 (en) 2004-03-12
US20060286087A1 (en) 2006-12-21
JP3857705B2 (ja) 2006-12-13
CA2391098A1 (en) 2002-01-17
ATE342964T1 (de) 2006-11-15
EP1332206B1 (en) 2006-10-18
ES2324727T3 (es) 2009-08-13
JP2004502456A (ja) 2004-01-29
WO2002004616A1 (en) 2002-01-17
BRPI0015533B8 (pt) 2021-05-25
CA2391098C (en) 2007-10-23
US6426208B1 (en) 2002-07-30
JP2005046154A (ja) 2005-02-24
CN1420924A (zh) 2003-05-28
MXPA02004742A (es) 2005-09-08
IL192078A0 (en) 2008-12-29
EP1792983A1 (en) 2007-06-06
JP2006312648A (ja) 2006-11-16
DE60031456T2 (de) 2007-08-23
DE60042137D1 (de) 2009-06-10
AU773663B2 (en) 2004-06-03
BR0015533A (pt) 2002-06-25
DE60031456D1 (de) 2006-11-30
HK1104836A1 (en) 2008-01-25
EP1332206A1 (en) 2003-08-06
IL149568A0 (en) 2002-11-10
CN101407796A (zh) 2009-04-15
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