Splicing Alteration TRIZ Cases

Splice-switching antisense oligonucleotides enable high-throughput screening of gene isoforms and link splice events to cell phenotypes.

PMOs target APP exons 7 and 8 to shift splicing toward APP695, reducing harmful isoforms linked to Alzheimer's progression.

Modified antisense oligonucleotides target progranulin pre-mRNA splice sites to boost the Exon1-Exon2 variant and restore protein levels.

Neutral PNA derivatives bind splice sites in pre-mRNA to drive exon skipping with strong cell uptake and lower toxicity than traditional oligonucleotides.

A deaminase-nickase fusion enables permanent exon skipping by editing splice sites while avoiding double-strand breaks and reducing off-target mutations.

Dual-targeting antisense oligonucleotides block UNC13A cryptic exon inclusion to restore protein expression in ALS and FTD.

An antisense oligonucleotide skips CEP290 exon 36 to bypass truncation, preserve the reading frame, and reduce retinal damage.

ABE8 base editing enables precise immune-cell gene changes that limit genomic rearrangements, immune suppression, and graft-versus-host risk.

Cell-penetrating peptide conjugates improve exon 44 antisense delivery and splicing correction to restore functional dystrophin in DMD.

Chemically modified ASOs target GRN pre-mRNA to raise brain progranulin levels while addressing delivery and off-target constraints.

Precise SMN2 base editing corrects the C6T splice regulator to restore stable SMN protein levels without transient dosing or overexpression toxicity.

Cationic and hydrophobic peptide conjugates improve oligonucleotide uptake and muscle distribution for stronger dystrophin restoration in DMD.

CRISPR guide RNAs target pathogenic SNPs to add poison exons or stop codons, lowering toxic protein RNA while preserving the wild-type allele.

Multiple orthogonal Cas9 proteins and guide RNAs enable simultaneous, independent gene regulation or editing across eukaryotic cells.

Targeted antisense oligomers block NMD-inducing SCN1A exon inclusion, restoring functional mRNA and boosting protein expression in Dravet Syndrome.

Anti-TfR1 antibody-oligonucleotide conjugates target muscle cells to reduce DMPK expression and correct splicing in myotonic dystrophy.

Internal nucleotide deletions break palindromic or repeated target motifs, improving antisense activity and easing oligonucleotide manufacture.

ENA-modified antisense oligonucleotides improve tau exon 10 splicing control, sustain 4R/3R ratio normalization, and reduce high-dose adverse events.

A bifunctional oligonucleotide binds alternative splice sites and recruits spliceosome components to regulate disease-linked RNA isoforms.

Splice-switching antisense oligonucleotides redirect RAGE pre-mRNA splicing to generate protective isoforms that counter harmful signaling.

CRISPR-Cas inserts a poison exon into the mutant HTT allele, triggering NMD to lower mHtt RNA and protein in Huntington's disease.

AAV-delivered inhibitory RNAs silence DMPK and disrupt CTG repeat expression, addressing DM1 treatment limits and improving disease modeling.

Shifting Fas splicing toward exon 6 skipping raises soluble Fas in T cells, improving survival, cytokine secretion, and target-cell killing.

Antisense oligonucleotides skip gamma-sarcoglycan exons 4, 5, or 6 to restore the reading frame and recover functional Mini-Gamma protein.