Self-immolating NanoSpermine (nSpm) as a novel treatment approach to restore normal spermine/spermidine ratios in Snyder-Robinson Syndrome (SRS) models.

2017SRS
Written By Million Dollar Bike Ride

Awardee: Robert Casero

Institution: John Hopkins University School of Medicine

Award Amount: $42,866

Funding Period: January 1, 2018 - December 31, 2018

Million Dollar Bike Ride
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CRISPR/Cas9 nuclease design to target allele-specific Collagen VI mutations in patients' fibroblasts
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Epigenetic Therapy to Treat Pitt-Hopkins Syndrome (PTHS)