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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).
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Nucleic Acid Sequence  |
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| ccacgacatatttttcuaca | |
