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dCas9+׻+Seelos˾ƶɭSLS-004ٴǰ

ʱ䣺2020-06-03 ȶȣ

Seelos Therapeutics˾ù˾ԤڸһΪSLS-004ɭѡٴǰо

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ɭParkinson’s diseasePDһֳϵͳ˶ƽΪ60ɭҪIJıԺʶͰdopamine, DAԪıɴ˶ʶͰԪε״DA̭²׷֢״ͨһ֫˶׾ۼԲ֫ٴҪΪֹ˶ǿֱƲ̬ϰ

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ɭķо۽α-ͻѰףα-synuclein, α-synα-synڴձһͻǰѰ״̬³ʿܵѰ׵ʽȺɲܵάѾ²ԪϸͬʶͰԪ̭ɭʣԪڷ·С壨Lewy bodyάα-ͻѰ·СҪα-synҩDZڵɭʱ

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SeelosSLS-004ƷеDNA׻תø3Aα-synucleinһضSCNA׻ŶSCNAļ׻α-synucleinıﱻͬʱ׼׻ϵͳCRISPR-dCas9༭ԶSNCAԾ΢α-synucleinı

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CRISPR-dCas9ջCRISPR-Cas9ܹgRNAָ¶ضıеʵֻϵµ

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Seelos³ϯִйRaj MehraʿǶα-synucleinصİеɭҪкܸߵȤSeelos׸ɭĻĿֵڴ

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ƾ֤SeelosչٴǰоSLS-004ͰԪϸɭ֢ļĸϸõظıSCNAĻα-synucleinˮƽͬʱSLS-004Ҳ

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       Seelos Therapeutics, Inc.һٴҩ˾ϵͳ߿ƷλŦԼSLS-004Seelosڲһɭ֢ƷѡSLS-007Ʒͬα-synucleinȺSeelos˾з߻SLS-002һֱȰͪƴӦϰ֢µɱΪSLS-006ɭߵҩͰ

ΪһͻϸƹҵCDMOЧ͹˾UG ṩעٴоͲҩٴ걨ƻٴƷҵGMPЧ漰ƷϸزͻͬʱӴҩ↑

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