COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
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United States of America
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Feb 13, 2025
app pub date -
May 9, 2022
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May 9, 2022
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Abstract
Compounds include at least one cyclic cell penetrating peptide (cCPP) conjugated to an antisense compound (AC). The AC modulates splicing of an RNA transcript. For example, the AC induces exon skipping. Exon skipping can result in down-regulation of expression or activity of a protein. Exon skipping may cause a frameshift in a resulting mRNA. The frameshift may result in a premature termination codon. The frameshift may result in nonsense mediated decay.
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