Splicing Alteration TRIZ Cases

Difficult pseudoexon splicing prediction is addressed with SSOs that promote mature mRNA incorporation and modulate target-protein expression.

This case uses IGS-guided RNA binding and trans-splicing to replace harmful APOE4 RNA in neurons with beneficial RNA.

This case shows how 5′-modified nucleosides and nucleotides support nuclease resistance while simplifying industrial production.

Chemical modifications improve DMD oligonucleotide stability and cell penetration, supporting exon skipping and dystrophin restoration.

This case combines anti-TfR1 antibody delivery with DMPK-targeting oligonucleotides to address ineffective DM1 therapies in muscle cells.

A guide RNA, RNA-binding domain, and repair template improve targeted trans-splicing specificity and efficiency for transient RNA repair.

This case uses exon 6-targeting SM-ASOs to modulate soluble IL7R while preserving membrane-bound IL7R in autoimmune and cancer therapy.

This case combines cell-penetrating peptides and carbohydrate targeting to improve intracellular oligonucleotide delivery to liver cells.

This case uses antisense oligonucleotides to suppress the intron 22 cryptic exon and increase correctly spliced CFTR mRNA.

Theophylline-dependent artificial riboswitches modulate pre-mRNA splicing by altering splice site accessibility upon ligand binding.

SaCas9 and SluCas9 proteins paired with guide RNAs excise small portions of the DMD gene to correct mutations while maintaining reading frame integrity.

Antisense oligonucleotides target glycogen synthase 1 mRNA to reduce enzyme levels in skeletal and cardiac muscles.

Allele-specific double stranded RNA targets mutant ataxin-3 mRNA via AAV9 vectors crossing the blood-brain barrier, preserving normal gene function.

Thiomorpholino oligonucleotides extend serum half-life and enable standard synthesis to treat muscular dystrophy.

Antisense oligomers target abundant poison exons in genes like ANKRD11 to bypass unproductive transcripts and restore functional protein levels.

Segmented diagnostic workflows detect intronic splice site mutations while antisense oligomers correct aberrant splicing to treat COL6-RD.

A synthetic nucleic acid expression system uses distinct promoters to drive separate pre-mRNA exons that trans-splice into a complete transcript.

Nanoparticles carrying deoxyribozymes and ligating enzymes splice RNA directly, avoiding DNA editing risks that cause cancer.

A splice-switching oligonucleotide binds to exon-intron junctions of the AluJb-LIN28B transcript to reduce oncogenic protein expression.

Attaching dual oligonucleotides to a cell penetrating peptide reduces cellular toxicity while maintaining delivery efficiency.

Short antisense oligonucleotides target the Exon V/Intron V junction of human HTR2C pre-mRNA to drive expression of the functional Vb splice isoform.

Covalent linkage with cell-penetrating peptides enhances delivery efficiency of antisense oligomers targeting PMP22 pre-mRNA.

Segmented CRISPR systems with specialized effector proteins resolve efficiency-reliability trade-offs in dystrophin gene detection and engineering.

Antisense oligonucleotides block the SMN2 ISS-N1 silencer, increasing exon 7 inclusion and restoring full-length SMN protein production.

Modified oligonucleotides bind fibronectin pre-mRNA introns to skip the EDA exon, reducing profibrotic protein levels.

Antisense oligonucleotides bind intron 7 to promote exon 7 inclusion, resolving insufficient SMN protein production in spinal muscular atrophy.

Adenine base editors modify dystrophin splice sites to restore the open reading frame without generating double-stranded breaks.

Chimeric tricyclonucleic acids with phosphodiester linkages resist enzymatic cleavage, reducing toxicity while maintaining high binding affinity.

Composite modified oligonucleotides resolve biostability versus cellular uptake trade-offs to restore dystrophin production in muscular dystrophy.

Combining specific antisense molecules into cocktails overcomes inconsistent exon skipping by targeting multiple splicing elements simultaneously.

Antisense oligonucleotides bind to myostatin pre-mRNA to induce exon skipping, avoiding viral vector safety risks while sustaining muscle mass.

3E10 antibody complexes protect antisense oligonucleotides from nuclease degradation while enabling targeted skeletal muscle delivery.

Antisense oligonucleotides target DYSF pseudoexons between exons 50 and 51, correcting abnormal splicing to restore functional dysferlin protein expression.

Antisense nucleic acids bind DYSF pre-mRNA to exclude pseudoexons, restoring functional dysferlin expression for muscle degeneration treatment.

dCas13 guides a trans-splicing donor construct to bind specific pre-mRNA targets, correcting deleterious mutations and enhancing gene expression.

Modified aptamer sequences enable precise gene regulation in eukaryotic cells, resolving interference issues from prokaryotic ligand sources.

Antisense oligonucleotides skip toxic exons in LRRK2 transcripts, reducing kinase activity while preserving cellular function.

Antisense nucleic acids target VCP exons to modulate protein expression, reducing neuropathology and improving muscle strength.

Triazine-cored amphiphilic cationic polymers form stable complexes with nucleic acids, reducing cytotoxicity while maintaining high transfection efficiency.

Dialkylamine side chains with phosphonic acid groups modify peptide nucleic acid monomers to enhance water solubility and cellular uptake.

Complementary oligonucleotides inhibit cryptic splicing in Usher transcripts, correcting protein expression for genetic therapy.

U7snRNA-mediated splice modulation via rAAV vectors overcomes walking ability plateaus in Duchenne Muscular Dystrophy caused by exon 2 duplication.

Cell-penetrating peptides conjugated to antisense compounds resolve low intracellular delivery efficiency and narrow tissue distribution.

Optical mapping measures action potential durations to identify ventricular tachycardia circuits, addressing repolarization heterogeneity.

Splice modulating oligonucleotides alter prolactin receptor pre-mRNA splicing patterns to generate tumor-suppressive protein forms.

Antisense oligonucleotides modulate tau mRNA splicing to reduce pathogenic protein levels in neurodegenerative disease treatment.

Methods identify and block aberrant pre-mRNA splicing by targeting specific regulatory hexamers, resolving misinterpretation of disease mutations.

Splicing-blocker oligonucleotides correct aberrant splicing defects to address underlying molecular mechanisms of cystic fibrosis.

Recombinant adeno-associated viruses deliver U7-based small nuclear RNAs to induce DUX4 exon skipping, reducing toxic full-length protein expression.

Antisense and RNA interference agents bind DUX4 transcripts to downregulate protein expression.