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

MDBRSRS

Feb 1

Written By Million Dollar Bike Ride

Awardee: Robert A. Casero, Jr

Institution: Johns Hopkins School of Medicine

Grant Amount: $69,318

MDBRSRS2019

Million Dollar Bike Ride

SETBP1 deficiency syndrome as an AKT modifer in human neurons

Rapid and unbiased in vivo screen for lead compounds that rescue STXBP1 dysfunction and subsequent in vitro validation in human neurons