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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)

Kushner FG, Hand M, Smith SC, King SB, Anderson JL, Antman EM, Bailey SR, Bates ER, Blankenship JC, Casey DE, Green LA, Jacobs AK, Hochman JS, Krumholz HM , Morrison DA, Ornato JP, Pearle DL, Peterson ED, Sloan MA, Whitlow PL, Williams DO