dCas9++Seelos˾ƶɭSLS-004ٴǰ
Seelos Therapeutics˾ù˾ԤڸһΪSLS-004ɭѡٴǰо
ɭParkinson’s diseasePDһֳϵͳ˶ƽΪ60ɭҪIJıԺʶͰdopamine, DAԪıɴ˶ʶͰԪε״DA̭²֢״ͨһ֫˶ۼԲ֫ٴҪΪֹ˶ǿֱƲ̬ϰ
ɭķо۽α-ͻѰףα-synuclein, α-synα-synڴձһͻǰѰ״̬³ʿܵѰʽȺɲܵάѾ²ԪϸͬʶͰԪ̭ɭʣԪڷ·С壨Lewy bodyάα-ͻѰ·СҪα-synҩDZڵɭʱ
CRISPR-dCas9ջCRISPR-Cas9ܹgRNAָ¶ضıеʵֻϵµ
Seelos³ϯִйRaj MehraʿǶα-synucleinصİеɭҪкܸߵȤSeelosɭĻĿֵڴ
ƾ֤SeelosչٴǰоSLS-004ͰԪϸɭ֢ļĸϸõظıSCNAĻα-synucleinˮƽͬʱSLS-004Ҳ
ΪһͻϸƹҵCDMOЧ˾UG ṩעٴоͲҩٴ걨ƻٴƷҵGMPЧ漰ƷϸزͻͬʱӴҩ↑
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