Dimethylspermine (Me2Spm) as a novel treatment approach to restore normal spermidine/spermine ratios in Snyder-Robinson Syndrome (SRS) models

MDBRSRS
Written by Million Dollar Bike Ride

Awardee: Robert A. Casero, Jr

Institution: Johns Hopkins School of Medicine

Grant Amount: $69,318

Million Dollar Bike Ride
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SETBP1 deficiency syndrome as an AKT modifer in human neurons
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Rapid and unbiased in vivo screen for lead compounds that rescue STXBP1 dysfunction and subsequent in vitro validation in human neurons