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

2017SRS

Jan 1

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

MDBR2017SRS

Million Dollar Bike Ride

CRISPR/Cas9 nuclease design to target allele-specific Collagen VI mutations in patients' fibroblasts

Epigenetic Therapy to Treat Pitt-Hopkins Syndrome (PTHS)