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elsunersen   Click here for help

GtoPdb Ligand ID: 14130

Synonyms: PRAX-222 | PRAX222
Compound class: Nucleic acid
Comment: Elsunersen (PRAX-222) is a 20-mer anti-sense oligonucleotide that is designed to selectively decrease expression of the gene for the voltage-gated sodium channel Nav1.2 (SCN2A). This is proposed as a mechanism to treat early onset developmental and epileptic encephalopathy (DEE) [1-3], that is driven by gain-of-function mutations in SCN2A. We include the linear nucleotide sequence in this entry, without the chemical modifications that are included in the administered version of elsunersen. A full descriptor is d([2'-O-(2-methoxyethyl)](m5rC-sp-m5rC-rA-5rC-rG-rA)-5C-sp-A-sp-T-p-A-p-T-p-T-p-T-p-T-p-T-p-m5C-sp-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA).
Targets where the ligand is described in the comment field
Target Comment
Nav1.2 The anti-sense oligonucleotide elsunersen is designed to reduce expression of gain-of-function SCN2A, as a proposed therapy for SCN2A-driven early-onset developmental and epileptic encephalopathy (DEE) [1].