Human HNF1-p65 transcription factors replace GAL4-VP16 in chimeric Notch receptors to reduce immunogenicity while preserving targeted gene expression.
Universal Th cell epitopes added to CMV VLPs boost helper T-cell activation and more consistent antibody responses, including in older adults.
Genotype-guided micro-dystrophin AAV therapy improves DMD patient selection while boosting muscle expression and limiting fibrosis.
Engineered NK-92 cells combine CAR, homing receptor, Fc receptor, and cytokine functions to improve tumor targeting and activity in suppressive microenvironments.
Targeted nanocarriers deliver transient gene editors to hematopoietic stem cells, avoiding viral complexity, cell sorting, and viability loss.
Targeted CD80 amino acid changes improve selective binding to CD28, PD-L1, and CTLA-4 for stronger immune modulation.
AAV-delivered GBA and PD-associated gene constructs target lysosomal dysfunction and α-synuclein to address refractory Parkinson's symptoms.
Targeted nucleic acid nanocarriers enable transient expression in hematopoietic stem cells to create lasting therapeutic changes without viral delivery.
Sequence optimization in an anti-VEGF rAAV cassette boosts expression for longer-lasting therapy with less immune response and irritation.
A PSCA-specific CAR uses tuned costimulation and an IgG4 Fc spacer to kill prostate tumors while limiting off-target activity and cytokine release.
Artificial tumor-surface epitopes give CAR-modified immune cells a uniform target, improving solid tumor recognition while limiting off-target toxicity.
Chemically regulated CD33 DARIC and CAR designs improve T cell persistence and control signaling to reduce toxicity and antigen-independent activation.
Engineered extracellular vesicles combine antigens, adjuvants, and targeting moieties to boost vaccine immune response and clinical efficacy.