CRISPR
KGI
Keck Graduate Institute (KGI) is exploring an investigational Cell Penetrating Peptide (CPP) UBE3A Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) approach to treating Angelman syndrome.
Funded
Discovery & Dev
Discovery & Dev
Pre-clinical
Phase 1
Phase 2
Phase 3
To patients
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FAST Pillar
Therapeutic Approach
A gene editing tool that utilizes a guide RNA (gRNA) to recognize a specific section of DNA or RNA and directs an enzyme (nuclease) to cut at a specific section in the DNA or RNA. Designing a CRISPR that affects the UBE3A-ATS could potentially allow the paternal UBE3A gene to be turned on. It could also be used to edit a mutation in a gene, known as base editing.
