Gene Therapy TRIZ Cases
- ADAMTS13 Transgene Truncation for AAV-Limited Gene Therapy
Selective ADAMTS13 domain retention fits AAV packaging limits while preserving autoregulation and sustained TTP control.
- Transgene Cassettes Using miRNA Targets for Cell-Specific Expression
miRNA target sequences linked to a transgene suppress expression in non-target cells, improving cell specificity and reducing off-target effects.
- HCII-Targeting Nucleic Acid Duplexes for Precise Gene Silencing
Partially complementary duplex nucleic acids inhibit HCII expression while balancing sequence specificity, stability, and targeted delivery.
- OR2AT4 Agonist Aptamers for Specific Receptor Activation
Nucleic acid aptamers specifically activate OR2AT4 to support wound healing, hair growth, and antiproliferative effects with better batch consistency.
- Retinal LNP-mRNA Composition for Even Photoreceptor Delivery
Ionizable lipid LNPs deliver mRNA evenly to RPE and photoreceptor cells at low doses, avoiding AAV size limits and reducing retinal toxicity.
- RNA Ribozyme Nucleic Acid Cleavage Without Cas Protein Burden
An RNA ribozyme from Group IIC introns enables sequence-specific DNA and RNA cleavage while avoiding Cas protein size, transfection, and immune issues.
- LEDGIN-Mediated HIV Integrase Retargeting for Functional Cure
By blocking LEDGF/p75-integrase binding, LEDGINs retarget HIV integration and keep latent reservoirs transcriptionally silent.