UCSF

The University of California San Francisco (UCSF) is investigating a novel Clustered Regularly Interspaced Short Palindromic Repeats Activation (CRISPRa) approach for gene upregulation for the genes other than UBE3A that are deleted in Deletion Angelman syndrome.

Discovery & DevPre-clinicalPhase 1Phase 2Phase 3To Patients

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About the Program

A gene modulation technology that utilizes a nuclease-deactivated Cas9 protein that binds to the target genomic region with the same efficiency as Cas9, but does not cut the DNA and instead can exert RNA-directed transcriptional control of the targeted gene with a goal to upregulate a specific gene.