SCN1ANAT-targeting antisense oligonucleotide

SCN1ANAT-targeting antisense oligonucleotide refers to a class of therapeutic agents, most notably represented by CAMP4 Therapeutics' CMP-SCN (formerly OPK88001 / CUR-1916, originally developed by OPKO Health), designed to treat Dravet syndrome. Dravet syndrome is a severe, haploinsufficient genetic epilepsy primarily caused by loss-of-function mutations in one allele of the SCN1A gene, which encodes the NaV1.1 voltage-gated sodium channel subunit alpha. This therapeutic approach utilizes an antisense oligonucleotide (ASO) to target and inhibit SCN1ANAT (SCN1A Natural Antisense Transcript), a long non-coding RNA that acts as an endogenous repressor of SCN1A transcription. By blocking this inhibitory transcript, the ASO upregulates transcription from the remaining wild-type SCN1A allele, restoring NaV1.1 protein levels and neuronal function, thereby reducing seizure frequency and severity.

Other names

SCN1A-NAT-targeting ASOSCN-1A-NAT-targeting ASOSCN 1A-NAT-targeting ASOSCN1A-NAT-targeting antisense oligonucleotideSCN-1A-NAT-targeting antisense oligonucleotideSCN 1A-NAT-targeting antisense oligonucleotideSCN1ANAT-targeting ASOSCN-1ANAT-targeting ASOSCN 1ANAT-targeting ASO