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American Epilepsy Society Annual Meeting: CMP-002: A Developmental Candidate Antisense Oligonucleotide Targeting a SYNGAP1 Regulatory RNA For The Potential Treatment of SYNGAP1-Related Disorders (Camp4 Therapeutics Corporation)

Public Technologies 10 Dec 2025
Nascent SYNGAP1 mRNA ... Total SYNGAP1 Nascent SYNGAP1 ... SYNGAP1 RNA following CMP-002 treatment ... •Treatment of patient iPSC-derived neurons carrying a heterozygous SYNGAP1 (K1185X) restored SYNGAP1 expression towards wild-type levels ... SYNGAP1 mRNA.
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