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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).
Classification Click here for help
Compound class Nucleic acid
Compound subclass Antisense oligonucleotide (ASO)
Target SCN2A mRNA
International Nonproprietary Names Click here for help
INN number INN
12610 elsunersen
Synonyms Click here for help
PRAX-222 | PRAX222
Database Links Click here for help
CAS Registry No. 2756001-75-7 (source: WHO INN record)
GtoPdb PubChem SID 518520757
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