Splicing Alteration TRIZ Cases
- Antisense Oligonucleotides Targeting H3.3 K27M Gliomas
Antisense oligonucleotides target mutant H3F3A mRNA to reduce tumor growth and promote neural stem cell differentiation.
- Antisense Oligomer Splicing Modulation for GAA Protein Restoration
Antisense oligomers hybridize to pre-mRNA regions to drive exon 2 inclusion and restore functional acid alpha-glucosidase protein levels.
- Inosine-Modified Oligonucleotides for DMD Exon Skipping
Inosine-containing oligonucleotides induce dystrophin synthesis while reducing immunogenicity and aggregation.
- Antisense Oligomer Splicing Modulation for Type VII Collagen
Antisense oligomers bind COL7A1 pre-mRNA splice junctions to exclude exon 80 and restore functional type VII collagen expression.
- Allele-Specific Splice Switching Oligonucleotides for Pseudoexon Incorporation
Allele-specific splice switching oligonucleotides promote pseudoexon incorporation into mature mRNA transcripts.
- Modulating APP Splicing to Reduce Aβ42 Production
Modified oligonucleotides block exon 17 splicing in the APP transcript, preventing gamma-secretase cleavage and reducing neurotoxic Aβ42 levels.
- AAV Vector Antisense Oligonucleotide Exon 53 Skipping
Modified antisense oligonucleotides in AAV vectors restore reading frames, reducing injection frequency and toxicity.
- Antisense Oligonucleotides Restore TDP-43 RNA Binding Function
Antisense oligonucleotides bind conserved TDP-43 binding sites on pre-mRNA transcripts, correcting aberrant splicing and restoring normal gene expression.
- Antisense Oligonucleotides Modulating CFTR RNA Splicing
Antisense oligonucleotides hybridize with CFTR RNA transcripts to modulate splicing, restoring functional protein levels despite persistent lung infections.