A representative report: ranked candidates, full chemistry and thermodynamics, off-target screening, and the reasoning behind each. Illustrative sample — not a real patient design.
This candidate targets the cryptic splice site introduced by the variant, with strong predicted binding affinity (Tm 62.4°C) and balanced GC content — minimising both instability and off-target promiscuity.
BLAST screening against the human transcriptome returned no high-risk homology. Low self-complementarity reduces the chance of hairpin formation that would compromise target engagement. The 2′-MOE phosphorothioate backbone matches the chemistry of approved splice-switching ASOs such as Spinraza.
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This report is an illustrative sample. Sequences and metrics are for demonstration only and do not represent a real patient design. All Pequliar outputs are computationally predicted candidates requiring independent experimental validation by qualified professionals.