Vectors TRIZ Cases
- Erythroid-Specific Promoter for Targeted Hemoglobinopathy Gene Expression
An erythroid-specific glycophorin A promoter confines transgene expression to red-cell lineage cells, improving hemoglobinopathy therapy safety.
- MBNL1 and RNAi Vector Delivery With Tissue Detargeting for Myotonic Dystrophy
Tissue-specific AAV delivery combines MBNL1 expression with RNA interference to suppress toxic repeat RNA while limiting cardiotoxicity.
- Multi-Target Functional Nucleic Acids for Protein Translation Upregulation
A single nucleic acid with multiple binding sites and SINE B2 or IRES elements boosts translation of several proteins for complex gene defects.
- Lentiviral Vector Insulators That Eliminate Cryptic Splice Sites
Modified HS4-derived insulators remove cryptic splice acceptor sites to prevent truncated transcripts and safer lentiviral gene therapy.
- Synthetic Liver-Specific Inducible Promoters for Controlled Gene Expression
A CAR-RXR-activated synthetic promoter enables liver-specific, dose-dependent gene expression while minimizing off-target activity.
- Dual-Inducer Gene Switch for Low-Leak Biologics Expression
A dual cumate coumermycin switch cuts leakiness and raises On/Off control, enabling stable mammalian cell lines for cytotoxic biologics.
- FXI Therapy for HFpEF Diastolic Dysfunction and Fibrosis
Liver-expressed FXI targets HFpEF-specific fibrosis, inflammation, and diastolic dysfunction through BMP-Smad1/5 pathway activation.
- Pancreatic HNF1A Gene Therapy for Durable MODY3 Glucose Control
AAV delivery of pancreas-specific HNF1A targets beta-cell dysfunction in MODY3 to improve glucose tolerance and reduce hyperglycemia.
- Regulatable Fusogenic HSV-1 for Selective Solid Tumor Killing
A tetracycline-regulated fusogenic HSV-1 boosts tumor-selective replication while limiting normal-cell toxicity and enhancing checkpoint therapy.
- Motor Neuron AAV Reprogramming to Delay ALS Degeneration
Motor neuron-specific AAV delivery of Isl1 and Lhx3 reactivates embryonic programs to resist ALS pathology and delay symptom onset.
- Genetically Modified NK-92 Cell Line for CD16-Mediated ADCC
Adding CD16 to NK-92 cells enables monoclonal antibodies to trigger ADCC while preserving their inherent cytolytic activity.
- Dumbbell Circular RNA Precursors for DNA-Splint-Free Ligation
A self-hybridizing dumbbell precursor enables T4 RNA ligase 2 circularization without DNA splints, simplifying purification and improving circRNA yield.
- 5′-UTR Sequence Design for Efficient mRNA Translation
Specific 5′-UTR motifs, reduced uridine content, and controlled secondary structure improve mRNA translation and protein yield.
- FOXP3–Helios Engineered T Cells for Stable Treg Expansion
Separate FOXP3 and Helios constructs generate eTregs that expand rapidly while retaining phenotype and limiting conventional T-cell contamination.
- Bacterial CRISPR Recording Cells for Non-Invasive Gut Assessment
Engineered bacterial cells convert transient gut RNA signals into stable CRISPR DNA spacers for non-invasive intestinal function assessment.
- Artificial Nucleic Acid Molecules With TOP-Gene UTRs for Stable Expression
TOP-gene 5′ UTRs and optimized coding sequences stabilize mRNA and improve protein expression without genomic integration.