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

Awardee: Alessandra Recchia

Institution: University of Modena and Reggio Emilia

Award Amount: $41,831

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

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Functional dissection of CRB1 homophilic interactions in Retinitis pigmentosa

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