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BMJ Open (2019) 9:e022188

Pipeline strategies include (i) AAV gene replacement for SLC52A2/A3 , (ii) brain-targeted delivery of active flavins, and (iii) small-molecule chaperones that stabilize misfolded transportersall of which will require biomarker-guided protocols and functional imaging endpoints to quantify long-term efficacy (19)

Solberg, O

Layer with other serums as needed

10.1038/sj.onc.1207105 258 RobergeS.RousselJ.AnderssonD

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