Splicing Alteration TRIZ Cases
- Splice Switching Oligonucleotides for Pseudoexon Incorporation
Difficult pseudoexon splicing prediction is addressed with SSOs that promote mature mRNA incorporation and modulate target-protein expression.
- APOE4 RNA Trans-Splicing Ribozyme for Specific Neuron Targeting
This case uses IGS-guided RNA binding and trans-splicing to replace harmful APOE4 RNA in neurons with beneficial RNA.
- 5′-Modified Nucleoside and Nucleotide Without Diastereomer Separation
This case shows how 5′-modified nucleosides and nucleotides support nuclease resistance while simplifying industrial production.
- Chirally Controlled DMD Oligonucleotides for Stable Exon Skipping
Chemical modifications improve DMD oligonucleotide stability and cell penetration, supporting exon skipping and dystrophin restoration.
- Dosing Anti-TfR1 Oligonucleotide Complexes for Muscle DMPK Reduction
This case combines anti-TfR1 antibody delivery with DMPK-targeting oligonucleotides to address ineffective DM1 therapies in muscle cells.
- CRISPR/Cas Targeted Trans-Splicing for Specific RNA Repair
A guide RNA, RNA-binding domain, and repair template improve targeted trans-splicing specificity and efficiency for transient RNA repair.
- Soluble IL7R Splice Modulation with Antisense Oligonucleotides
This case uses exon 6-targeting SM-ASOs to modulate soluble IL7R while preserving membrane-bound IL7R in autoimmune and cancer therapy.
- CPP–Carbohydrate Conjugates for Hepatocyte Oligonucleotide Targeting
This case combines cell-penetrating peptides and carbohydrate targeting to improve intracellular oligonucleotide delivery to liver cells.
- CFTR Splicing Modulation to Suppress Intron 22 Cryptic Exon
This case uses antisense oligonucleotides to suppress the intron 22 cryptic exon and increase correctly spliced CFTR mRNA.