HUE032074T2 - Aktív, nagymértékben foszforilált, emberi liszoszomális szulfatáz enzimek elõállítása és alkalmazása - Google Patents
Aktív, nagymértékben foszforilált, emberi liszoszomális szulfatáz enzimek elõállítása és alkalmazása Download PDFInfo
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- HUE032074T2 HUE032074T2 HUE09702074A HUE09702074A HUE032074T2 HU E032074 T2 HUE032074 T2 HU E032074T2 HU E09702074 A HUE09702074 A HU E09702074A HU E09702074 A HUE09702074 A HU E09702074A HU E032074 T2 HUE032074 T2 HU E032074T2
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- galns
- enzyme
- lake
- sulfatase
- lysosomal
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Claims (2)
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VÍC :gjÿ tó* s; C :F' v>; -ÖSs ^ ^ ^ XI ^ ’<: F g iá ® p ' ' tó B -tó: S ® :» tó ä é -í*i· 8 í * 1 I 1- I I: s S i 1 J I Ï 8: f i € * Ç f Ψ í if S. s s: i * I ? I r I = 50. A ?. igénypont szerinti készítmény az oit meghatározott alkalmazásra vagy a 8. igéttypom szerlnu alkalmazás. ahol a CiALMS enzim redukáló körolmények között vág»«» SOS·· F A GO. voran Coenumie Slue îVÿ-· ’|5:S^ifíl0i|;:í>íí^í!i^i^ ϊΐ·ίI, έί:«>όϊ>ί·;;?ί-:lá.íMií;íí proteinek. legalább 90Η-3 ||||||||||||||||, j!!Í:'''::|||||Í. t. î. kés/.itmény nz ctt :^:::^ÎÎ;^-iiiiiiiiii^ !!l eiéoypomok bálibbΙΑ:5í ΐίΐ5¾¾ iíH|||.:GÁl:iP:S ehzlm IBIpil^ií&íáíjSít levő tisztein legalább Am ä Cso-fotmilgilcmne (FCUy) kö|yéÍÍtíl 1:2; A 7-50, igénypontok bármelyike szerint! készítmény az. ott megbatározott alkalmazásra vagy ah-ltl igénypontok-bármelyike szerinti alkalmazás, ahol a GAIMS enzim S3, pozickiíabtt« íévö -tisztem J.egaí-áb.b.-9Uk«* á.Cö-íomulgHcinné (FQIy) -konvertált:.· 13. A 7-12. igénypontok bármelyike szerinti készítmény az-ott meghatározott .alkat ntázásra vagy a 1M2. Igénypontok bánttciyíke szerinti alkalmam, ahol a kezeié?; hatásosságát az MÍ>.S iVavban vagy .Montul» A szindróntában szenvedd alanyban a ksratán-szatiat vizelettel-tÖrténiVkiválasZtáaán&k mérésével határozzak öreg. aha! a* MFS IVa-ban vagy Morcom Ä szbidmmábast szenvedő alany vizeleten mért KS-sxntbéi ittíroíál alany vízek'tben mert ΗΛ .-cmtH'rtd e* vagy MFh. iVa-ban vagy Morquio A szindrómába» -szenvedő, kezeletlen alany vizeletben'»}«« K^szmíjéval vagy a« azonos alany GALMS~enzimnse|:.td«éná-lctÍzeiéat megeJ&riie« a vizeletben mén KS-srJntjévcl hasonlítjuk össze, vagy ahoi a kezelés hatásosságát az alany fttnkekmáíis vizsgálatával tárástesztekkel, lépcsőzéssé! mért állóképesség vagy polmonáliaAesptratorifcua funkció alapján határozzuk meg, ahol adok esetben a járásieszt « 6'vágy ' 12 'pere alatt sétál· 'távolságot ntéo ystgyl!! :$hb:|:Í;$á^ .pöSpilliält: meglesi lépcsők s/asnüt méri vagy ahol a puimonáii.Vre-piratorrki.i!·: inrskdot korökhis funkció íeehokardmcram} vagy putmanálss fenkció íl'VC, FFV llkhiy «íiííiíSa^^mléiä|S|íiíáís M. A 13. igénypont szerinti készítmény az ott meghatározott alkalmazásra. vagy tt 1:1 igénypont szentül alkalmazás, ahol GAUNS-enzimtuel történő kezelést követően a vizeletben mért KS csökkenése nagyobb, miot 201(, vagy ahol tiALNS-enstmmel történő kezdést követően a vizeletben mert KS csökkenése nagyobb, mint lil!
- 15, Az t-<S, igénypontok bármelyike szerinti O A LNS^eîtzimkészJîtnény. a 7-14. igénypontok bármelyike szerinti készítmény az ott meghatározót} alkalmazásra vagy a l-M. igénypontok bármelyike szét inti alkalmazás, ahol a sebes irányítószígnái tartalmaz, osont iíáavlrópeptidei.
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US8026083B2 (en) | 2007-04-03 | 2011-09-27 | Oxyrane Uk Limited | Yarrowia lipolytica and Pichia pastoris HAC1 nucleic acids |
CN105274072A (zh) * | 2008-01-18 | 2016-01-27 | 生物马林药物股份有限公司 | 制备活性高度磷酸化人溶酶体硫酸酯酶及其应用 |
CA2775938C (en) | 2009-09-29 | 2021-08-10 | Oxyrane Uk Limited | Hydrolysis of mannose-1-phospho-6-mannose linkage to phospho-6-mannose |
JP2013511272A (ja) | 2009-11-19 | 2013-04-04 | オキシレイン ユーケー リミテッド | 哺乳類様複合n−グリカンを生成する酵母株 |
AR082319A1 (es) * | 2010-07-22 | 2012-11-28 | Biomarin Pharm Inc | Produccion de una n-acetilgalactosamina-6-sulfatasa humana activa altamente fosforilada y sus usos |
EP2622088A2 (en) | 2010-09-29 | 2013-08-07 | Oxyrane UK Limited | Mannosidases capable of uncapping mannose-1-phospho-6-mannose linkages and demannosylating phosphorylated n-glycans and methods of facilitating mammalian cellular uptake of glycoproteins |
CN103261432A (zh) | 2010-09-29 | 2013-08-21 | 奥克西雷恩英国有限公司 | 磷酸化的n-聚糖的脱甘露糖基化 |
US20130236442A1 (en) * | 2010-11-12 | 2013-09-12 | Green Cross Corporation | Iduronate-2-sulfatase and use thereof |
US9206401B2 (en) * | 2010-12-22 | 2015-12-08 | Fondazione Telethon | Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses |
TW201307563A (zh) | 2011-05-19 | 2013-02-16 | Shire Human Genetic Therapies | 純化乙醯肝素-n-硫酸酯酶之方法 |
US20160145589A1 (en) | 2011-06-24 | 2016-05-26 | Green Cross Corporation | Composition and formulation comprising recombinant human iduronate-2-sulfatase and preparation method thereof |
KR20190114003A (ko) | 2012-03-15 | 2019-10-08 | 옥시레인 유케이 리미티드 | 폼페병의 치료를 위한 방법 및 물질 |
WO2013148323A1 (en) * | 2012-03-30 | 2013-10-03 | Shire Human Genetic Therapies | Methods of analyzing and preparing protein compositions |
KR101380740B1 (ko) * | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
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